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Juxtapid lomitapide mesylate 20 mg Capsule, 28-count — NDC 76431-0120-01 package photo
Label image from the product's FDA listing (DailyMed) — may show a different pack size or an older label revision.

Juxtapid lomitapide mesylate 20 mg Capsule, 28-count — NDC 76431-120-01 (Billing 76431-0120-01)

by Amryt Pharmaceuticals DAC · 28 CAPSULE in 1 BOTTLE

This is a package of 28 capsules of Juxtapid lomitapide mesylate 20 mg Capsule from Amryt Pharmaceuticals DAC, marketed since Jan 2013 and currently FDA-listed, this package's marketing is listed to end Oct 2026. It is this product's only package size.

NDC 76431-0120-01
🏷️ FDA NDC (as labeled) 76431-120-01 billing pads the product segment with a zero
Rx only Brand On market Non-controlled 🛡 REMS ⇄ Compare with another NDC
🗂️ FDA directory synced Oct 1, 2026 · this listing last changed Jul 24, 2026 · sources: openFDA · FDA label (DailyMed) · FDA Orange & Purple Book · First Databank · CMS NADAC, ASP, Medicare & Medicaid · RxNorm
📋 All sources & update times →

This page is curated, coded, and maintained by

Compiled from public FDA, CMS, DailyMed, NADAC, and related drug data sources. Editorial policy

NDC database record

One package, one record: these facts belong to NDC 76431-120-01 alone.

Record
FDA NDC Directory package listing · Human prescription drug
Code segments
76431 labeler · 120 product · 01 package
Package marketed since
Jan 28, 2013
Package marketing ended
Oct 25, 2026
Sample package
No — commercial package
Billing quantity
28 EA per package
Barcode (UPC-A, from the NDC)
3 7643112001 4
FDA record last changed
Jul 24, 2026

Identity & classification

Regulatory identifiers FDA, NLM and CMS codes for this package

FDA NDC (as labeled) 76431-120-01
Product NDC 76431-120
11-digit billing NDC 76431012001
NCPDP billing unit EA — each (per item)
RxCUI 1364484, 1364490, 1364492, 1364494, 1364496, 1364498, 1648770, 1648772
UNII X4S83CP54E
Application # NDA203858
SPL Set ID e4c45bb5-15f4-437e-ab98-a649b3676d14
Established class (EPC) Microsomal Triglyceride Transfer Protein Inhibitor
Mechanism of action Cytochrome P450 3A4 Inhibitors; Microsomal Triglyceride Transfer Protein Inhibitors; P-Glycoprotein Inhibitors
DEA schedule Non-controlled
Marketing category NDA
Marketing status On market
FDA listing status Listed (active directory)
Marketing start 2013-01-03
Marketing end 2026-10-25
Route ORAL
Dosage form CAPSULE
Substance LOMITAPIDE MESYLATE

Drug-database identifiers Medi-Span GPI and First Databank GCN / HICL / AHFS classification

GPI-14 39480050200140
GPI class Juxtapid
GCN Seq No 070395
GCN 33913
HICL code 039883
Ingredient (HICL) Lomitapide Mesylate
HIC1 code M
Therapeutic class — broad (HIC1) Blood
HIC2 code M4
Therapeutic class — intermediate (HIC2) Affect Blood Lipids/Sugar/Amino Acids
HIC3 code M4Q
Therapeutic class — specific (HIC3) Antihyperlipidemic - Mtp Inhibitor
AHFS code 24:06.22.00
AHFS class Mtp Protein Inhibitors
FDB label name JUXTAPID 20 MG CAPSULE
FDB brand name Juxtapid
Legend status F — Federal legend — prescription drug or device
Quick answers
  • GSN (GCN sequence number): 070395
  • GCN: 33913
  • GPI-14 (Medi-Span): 39480050200140
  • HICL (First Databank): 039883
  • AHFS class code: 24:06.22.00
  • RxCUI (RxNorm): 1364484
Why two NDCs? The FDA registers this code as 76431-120-01 — a 5-3-2 layout, and that's what's printed on the package and shown on DailyMed. For insurance claims, every NDC is standardized to a uniform 11-digit 5-4-2 format by adding a zero to the product segment → 76431-0120-01. Same drug, same package — only the format differs.
Where does this data come from?
Identifiers from the FDA openFDA NDC Directory and Structured Product Labeling; RxCUI from RxNorm (NLM); GPI from Medi-Span; GCN / HIC / AHFS / legend from First Databank.

RxNorm drug class

This medicine belongs to the Microsomal Triglyceride Transfer Protein Inhibitor class.

Pharmacologic class Microsomal Triglyceride Transfer Protein Inhibitor
Drug family (ATC) Other lipid modifying agents
How it works Cytochrome P450 3A4 Inhibitors, P-Glycoprotein Inhibitors, Microsomal Triglyceride Transfer Protein Inhibitors
Where does this data come from?
Therapeutic classes from RxNorm RxClass (U.S. National Library of Medicine) — Established Pharmacologic Class (FDA), ATC drug family (WHO) and mechanism of action, matched by this product’s RxCUI.

Clinical

Label name JUXTAPID 20 MG CAPSULE Ingredient Lomitapide Mesylate
📖 What it is MedlinePlus · NLM

Lomitapide is used to lower cholesterol (a fat-like substance that can build up and clog blood vessels causing heart attacks or strokes or other health problems) in people that have homozygous familial hypercholesterolemia (HoFH; an inherited condition in which cholesterol cannot be removed from the body normally). Lomitapide is in a class of medications called cholesterol-lowering medications. It works by slowing the production of cholesterol in the body to decrease the amount of cholesterol that may build up on the walls of the arteries and block blood flow to the heart, brain, and other par...

Read the full MedlinePlus article ↗
📗 Our plain-language guide HelloPharmacist
  • It’s for homozygous familial hypercholesterolemia (HoFH), a rare inherited condition causing very high LDL cholesterol. You take it along with a low-fat diet and other cholesterol...
  • Take the capsule by mouth exactly as your prescriber directs. Stick to your low-fat diet and take your vitamin E and essential fatty acid supplements every day. Avoid grapefruit ju...
  • Stomach upset is very common: diarrhea, nausea, indigestion, vomiting and belly pain. A low-fat diet helps. If diarrhea is severe or you feel lightheaded, are urinating less or fee...
  • Lomitapide can raise liver enzymes and build up fat in the liver. Regular liver tests let your doctor lower the dose or stop the medicine early if there’s a problem. It’s also why...
📖 Read our full Lomitapide guide →

Supplement & herbal interactions

🌿 Drug–supplement interaction checker →
Where does this data come from?
Plain-language summary from MedlinePlus (U.S. National Library of Medicine); supplement & herbal interactions and nutrient depletion data from the Natural Medicines database; our full guide is HelloPharmacist editorial content.

Pricing

A drug doesn't have one price. Each row is a different public payment system, and none is what you'd pay at the counter — that depends on your insurance. The ⓘ on each row explains what it measures.

Price systemPer eachPer package
Retail pharmacies payNADAC · weekly Not in the retail survey — common for institutional, discontinued, or low-volume packs.
Medicaid paysCMS SDUD · 12 mo No recent Medicaid claims on file for this NDC — rare and low-volume NDCs are suppressed in the public data.
Medicare drug plans payPart D · Q2 2026 $2,068.50 $57,918.03 / 28 capsules
Where does this data come from?
NADAC (National Average Drug Acquisition Cost) is the CMS weekly pharmacy-acquisition-cost survey — what pharmacies pay. ASP (Average Sales Price) is the CMS Medicare Part B drug-payment file, published quarterly. Medicaid pays is computed by us from CMS State Drug Utilization Data (total reimbursed ÷ units, trailing 12 months) — gross of rebates and inclusive of dispensing fees, so it reflects what Medicaid paid, not an acquisition cost. Medicare drug plans pay is the median negotiated point-of-sale unit cost across plans listing this NDC in the CMS quarterly Prescription Drug Plan pricing files, before rebates. The VA pays is the federal contract price (FSS, and the statutory Big 4 ceiling where listed) from the VA National Acquisition Center pharmaceutical price file. All are free public government data; each measures a different payer, so the figures are not directly comparable.

Packaging — all sizes for this product

Package NDCDescription Marketing startMarketing endStatus
76431-0120-01 You're viewing this Main listing 28 CAPSULE in 1 BOTTLE 2013-01-28 Oct 25, 2026 Active

Therapeutic equivalents

ProductLabelerPackNADAC/unitTEStatusPrice vs. this
Juxtapid 20 mg 10122-0420-28 Chiesi 28 capsules — — FDA listed —
Juxtapid 20 mgthis 76431-0120-01 Amryt 28 capsules — — FDA listed —
Juxtapid 20 mg 75929-0027-25 Pharma 28 capsules — — Discontinued —
Juxtapid 20 mg 75929-0243-14 Pharma 28 capsules — — Discontinued —
About this product: this is the brand-name version. We did not find an FDA-approved generic match for this exact strength, form and route.
Where does this data come from?
Equivalents are other NDCs of the same ingredient, form and route from the openFDA NDC Directory, ranked least-expensive-first by NADAC. Therapeutic-equivalence (AB) ratings come from the FDA Orange Book; biologics use the FDA Purple Book for biosimilar & interchangeable status.

Availability & generic status

🏛️
2015
First FDA approval
Apr 2015
📍
2026
Currently FDA-listed
11 years listed
🛡️
2041
Latest patent/protection listed
not a guaranteed launch date
🔒No FDA-approved generic found

We did not find an FDA-approved generic match for this exact strength, form and route. Patent/protection dates below may affect future generic timing.

🛡️ Latest patent/protection date listed: FDA patent/protection data lists protections through Jul 2041. This may affect when a full generic version becomes widely available, but it is not a guaranteed launch date.
📅 FDA approved Apr 23, 2015 RLD RS ⏳ ~14.8 yr to latest listed protection

Why the date isn’t exact: Generic timing can change because patents may be challenged, settled, licensed, added, removed, or worked around with a narrower label — and FDA approval does not always mean a pharmacy can get the generic today.

Patents & exclusivity — FDA Orange Book
US 7932268 — method of use (U-1316)
US 7932268 — method of use (U-1316)
US 7932268 — method of use (U-1316)
US 7932268 — method of use (U-1316)
US 7932268 — method of use (U-1316)
US 12472172 — method of use (U-4438)
US 12472172 — method of use (U-4438)
US 12472172 — method of use (U-4438)
US 12472172 — method of use (U-4438)
US 12472172 — method of use (U-4438)
US 7932268 — method of use (U-1316)
US 7932268 — method of use (U-1316)
Exclusivity NPP
Exclusivity NPP
Exclusivity NPP
Exclusivity NPP
Exclusivity NPP
Exclusivity NS
2015 2017 2019 2021 2023 2025 2027 2029 2031 2033 2035 2037 2039 2041
Today
LOE
Substance patent Formulation patent Method-of-use patent Exclusivity Pediatric +6mo
🏛️FDA exclusivity
FDA-granted marketing protection. It’s separate from patents and may be shorter than patent protection.
🧪Product / substance patents
Patents covering the active ingredient, product, formulation, or related drug features.
🎯Method-of-use patents
Patents covering specific approved uses. These can sometimes be carved out with a “skinny label,” but not always.
🛈 What do these terms mean?
Patent
Legal protection listed in the Orange Book that may delay generic approval or launch. Issued by the U.S. Patent & Trademark Office.
Substance patent
Covers the active drug molecule itself — the hardest to design around. A generic generally can’t launch until it expires.
Formulation (product) patent
Covers a specific formulation or dosage form. A generic can sometimes work around it with a different formulation.
Method-of-use patent
A patent covering one specific approved use of the drug — not necessarily the whole molecule. A generic can sometimes launch with a “skinny label” that carves out the protected use and keeps the others.
Skinny label
A generic label that omits a still-patented use when the FDA allows it — letting a generic reach the market for the unprotected uses.
Exclusivity
FDA-granted marketing protection, separate from patents — e.g. 5-yr new chemical entity, 7-yr orphan drug, or a +6-month pediatric extension.
Paragraph IV
A generic applicant’s formal challenge to a listed patent. It can potentially lead to earlier generic entry, but often involves litigation or a settlement.
RLD / RS
Reference Listed Drug — the brand product the FDA uses as the reference for generic applications. Reference Standard — the product the FDA expects generics to compare against in bioequivalence testing.
TE / AB rating
FDA therapeutic-equivalence rating. An AB rating generally means the FDA considers a generic therapeutically equivalent to — and substitutable for — the brand.
LOE (loss of exclusivity)
The latest patent or exclusivity currently listed — the loss-of-exclusivity / latest-listed-protection date shown on this page. Paragraph-IV challenges and settlements can move the real date earlier; FDA approval and a manufacturer’s decision to market can move it later.

Built from the FDA Orange Book. The bars above are scaled to each protection’s expiry; the red LOE marker is the last one to lapse.

Listed patents (12)
PatentTypeUse codeExpires
US 7932268 ↗ Method of use U-1316 Aug 19, 2027
US 7932268 ↗ Method of use U-1316 Aug 19, 2027
US 7932268 ↗ Method of use U-1316 Aug 19, 2027
US 7932268 ↗ Method of use U-1316 Aug 19, 2027
US 7932268 ↗ Method of use U-1316 Aug 19, 2027
US 12472172 ↗ Method of use U-4438 Jul 29, 2041
US 12472172 ↗ Method of use U-4438 Jul 29, 2041
US 12472172 ↗ Method of use U-4438 Jul 29, 2041
US 12472172 ↗ Method of use U-4438 Jul 29, 2041
US 12472172 ↗ Method of use U-4438 Jul 29, 2041
US 7932268 ↗ Method of use U-1316 Aug 19, 2027
US 7932268 ↗ Method of use U-1316 Aug 19, 2027
FDA exclusivity
CodeWhat it grantsExpires
NPPNew Patient PopulationFeb 25, 2029
NPPNew Patient PopulationFeb 25, 2029
NPPNew Patient PopulationFeb 25, 2029
NPPNew Patient PopulationFeb 25, 2029
NPPNew Patient PopulationFeb 25, 2029
NSNew StrengthFeb 25, 2029
Common questions
Is there a generic version of JUXTAPID 20 MG CAPSULE?
No FDA-approved generic equivalent is currently listed in the FDA Orange Book for JUXTAPID 20 MG CAPSULE. Based on the patents and exclusivity currently listed, the Orange Book estimate is that full-label generic entry may be delayed until Jul 2041 — an estimate, not a guaranteed launch date.
The FDA approved a generic — why can’t I get it at my pharmacy yet?
FDA approval and pharmacy availability are two different things. The FDA can approve a generic years before it actually reaches pharmacies, because the brand company may still hold patents or have a settlement that delays the launch. A manufacturer also has to choose to make and sell it, and have supply ready. So a drug can be “FDA-approved generic exists” and still be brand-only at the counter today.
Why do different websites show different generic release dates?
Generic availability is not based on one single date. Some sources use the first exclusivity expiration, some use the last product patent, and others use the latest method-of-use patent. Patent challenges, settlements, licenses, and label carve-outs can also change the real-world launch date. This page shows the underlying Orange Book dates so you can see why estimates may differ.
What does “FDA listed” mean?
It means the product appears in the FDA’s official NDC directory. That’s a good sign a product exists and is intended for the U.S. market, but on its own it does not confirm a pharmacy can fill it today. Where we have recent retail pricing data (NADAC) for a product, we label it “Availability likely” instead.
What does a patent or protection date mean here?
It’s the latest date currently listed in the FDA Orange Book for a patent or exclusivity on the brand product. It can affect when a full generic version becomes widely available — but it is not a guaranteed generic launch date. Generics sometimes arrive earlier (through a settlement or patent challenge) or later (a manufacturer still has to make and sell one).
What does “current Orange Book estimate” mean?
It means we are using the latest patent and exclusivity dates currently listed in the FDA Orange Book. It is not a guaranteed launch date.
Can a generic come out before the last patent expires?
Sometimes. A generic company may challenge a patent, settle with the brand manufacturer, receive a license, or obtain approval with a narrower label that avoids a patented use. In other cases, the last listed protection may delay full-label generic competition.
Can a generic come out after the listed dates?
Yes. Even after patents or exclusivity expire, a generic still needs FDA approval and a manufacturer must choose to market it. Supply, litigation, business decisions, or regulatory issues can delay actual availability.
What is the difference between patents and exclusivity?
Patents are legal protections usually issued by the U.S. Patent and Trademark Office. FDA exclusivity is marketing protection granted by the FDA. They are separate, and either one can affect generic timing.
Why are there multiple patent dates?
One drug can have several patents covering different things: the active ingredient, a formulation, a manufacturing process, or a specific approved use. That is why a page may show several expiration dates instead of one simple generic date.
Built from FDA Orange Book patent and exclusivity data. Dates are refreshed from public FDA data when available; the marker is max(latest patent expiry, latest exclusivity expiry). Paragraph-IV settlements and first-filer 180-day exclusivity can shift the real date; a method-of-use patent may allow an earlier skinny-label generic for non-protected indications. Generic launch timing is an estimate, not a guarantee.
Where does this data come from?
Patents and exclusivity from the FDA Orange Book (small-molecule drugs), refreshed from public FDA data. Generic launch timing is an estimate, not a guarantee.

What it looks like

Color Orange / White / Yellow
ShapeCapsule
ImprintA733;30mg
Size22 mm
ScoringNot scored
One label can cover several strengths, so colors may be combined — always confirm a loose pill against the dispensed prescription label or a pharmacist.
Where does this data come from?
Physical description (imprint, shape, color, scoring, coating) from this product’s FDA Structured Product Labeling (SPL), mirrored from DailyMed / openFDA.

Inactive Ingredients / Excipients

Inactive ingredients, also called excipients, are components of the drug product other than the active ingredient. They may include fillers, dyes, coatings, preservatives, flavors, or other formulation ingredients.

A current SPL was checked, but it does not contain a structured or narrative inactive-ingredient list for this product. This does not mean the product has no inactive ingredients.
Where does this data come from?
Source: official FDA Structured Product Labeling (SPL) via DailyMed and the openFDA label index. Structured IACT rows and label-wide narrative are kept separate; availability and product-level specificity depend on the submitted label.

Inactive ingredient FAQ

Are inactive ingredients the same for every manufacturer?
No. Inactive ingredients can differ by manufacturer, dosage form, strength, and package / product version.
Why might an inactive ingredient be missing?
Some SPLs do not provide a complete structured inactive-ingredient list, and older or unusual labels may only include the information in narrative text.
Can inactive ingredients matter?
Yes. They can matter for allergies, intolerances, dyes, gluten / lactose concerns, preservatives, and formulation differences — but confirm with a pharmacist or the manufacturer when it’s clinically important.

Manufacturer & labeler

LabelerAmryt Pharmaceuticals DAC
Application holderCHIESI FARMACEUTICI SPA
FDA applicationNDA203858 (NDA)
Labeler code76431
First marketedJan 2013
Product typeHuman Prescription Drug
Portfolio3 products on file

More NDCs from Amryt Pharmaceuticals DAC labeler code 76431

The labeler markets the product; the application holder owns the FDA approval. They’re often the same company but can differ (e.g. a repackager or an authorized generic). A mailing address / phone appears here when the manufacturer includes it in the product’s FDA label (not all do).
Where does this data come from?
Labeler, application holder and registered establishment from the FDA openFDA NDC Directory and Drugs@FDA; address/contact from the product’s FDA label.

Full prescribing information FDA SPL

The complete FDA label for this product — the official prescribing information, verbatim, section by section. Very long sections are excerpted here and marked; the full text is on DailyMed (linked in the sources below). Jump with a chip, search within the label, or expand everything.
🚨 Boxed Warning 🎯 Indications and Usage ⏱️ Dosage and Administration 💊 Dosage Forms and Strengths ⛔ Contraindications ⚠️ Warnings and Cautions 🤒 Adverse Reactions 🔄 Drug Interactions 👥 Use in Specific Populations 🤰 Pregnancy 🧒 Pediatric Use 🧓 Geriatric Use 🆘 Overdosage 🧬 Clinical Pharmacology 🧬 Mechanism of Action 📦 How Supplied / Storage and Handling 📦 Storage and Handling 📋 Description 💬 Information for Patients 💬 Medication Guide 🧬 Pharmacokinetics 🧬 Pharmacodynamics 🔬 Clinical Studies 🧪 Nonclinical Toxicology 📄 Carcinogenesis, Mutagenesis, Impairment of Fertility 📄 Package Label / Principal Display Panel
🚨 Boxed Warning ~2 min read ▾

WARNING: RISK OF HEPATOTOXICITY JUXTAPID can cause elevations in transaminases. In the JUXTAPID clinical trial, 10 (34%) of the 29 patients treated with JUXTAPID had at least one elevation in alanine aminotransferase (ALT) or aspartate aminotransferase (AST) ≥3× upper limit of normal (ULN). There were no concomitant clinically meaningful elevations of total bilirubin, international normalized ratio (INR), or alkaline phosphatase [see Warnings and Precautions (5.1) ].

JUXTAPID also increases hepatic fat, with or without concomitant increases in transaminases. The median absolute increase in hepatic fat was 6% after both 26 and 78 weeks of treatment, from 1% at baseline, measured by magnetic resonance spectroscopy. Hepatic steatosis associated with JUXTAPID treatment may be a risk factor for progressive liver disease, including steatohepatitis and cirrhosis [see Warnings and Precautions (5.1) ].

Measure ALT, AST, alkaline phosphatase, and total bilirubin before initiating treatment and then ALT and AST regularly as recommended. During treatment, adjust the dose of JUXTAPID if the ALT or AST are ≥3× ULN. Discontinue JUXTAPID for clinically significant liver toxicity [ see Dosage and Administration (2.4) and Warnings and Precautions (5.1) ].

Because of the risk of hepatotoxicity, JUXTAPID is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the JUXTAPID REMS Program [see Warnings and Precautions (5.2) ]. Prescribe JUXTAPID only to patients with a clinical or laboratory diagnosis consistent with HoFH. The safety and effectiveness of JUXTAPID have not been established in patients with hypercholesterolemia who do not have HoFH [see Indications and Usage (1) ] .

WARNING: RISK OF HEPATOTOXICITY See full prescribing information for complete boxed warning. JUXTAPID can cause elevations in transaminases ( 5.1 ). Measure alanine and aspartate aminotransferases (ALT, AST), alkaline phosphatase, and total bilirubin before initiating treatment and then ALT and AST regularly as recommended ( 2.4 , 5.1 ).

During treatment, adjust the dose of JUXTAPID if the ALT or AST is ≥3 times the upper limit of normal (ULN) ( 2.4 , 5.1 ). Discontinue JUXTAPID for clinically significant liver toxicity ( 2.4 , 5.1 ). JUXTAPID increases hepatic fat (hepatic steatosis) with or without concomitant increases in transaminases ( 5.1 ).

Hepatic steatosis associated with JUXTAPID may be a risk factor for progressive liver disease, including steatohepatitis and cirrhosis ( 5.1 ). Because of the risk of hepatotoxicity, JUXTAPID is available only through a restricted program called the JUXTAPID REMS Program ( 5.2 ). Prescribe JUXTAPID only to patients with a clinical or laboratory diagnosis consistent with HoFH.

The safety and effectiveness of JUXTAPID have not been established in patients with hypercholesterolemia who do not have HoFH ( 1 ).

🎯 Indications and Usage 195 words ▾

1 INDICATIONS AND USAGE JUXTAPID is a microsomal triglyceride transfer protein inhibitor indicated as an adjunct to a low-fat diet and other lipid-lowering treatments, including LDL apheresis where available, to reduce low-density lipoprotein cholesterol (LDL-C), total cholesterol (TC), apolipoprotein B (apo B), and non-high-density lipoprotein cholesterol (non-HDL-C) in patients with homozygous familial hypercholesterolemia (HoFH) ( 1 ). Limitations of Use The safety and effectiveness of JUXTAPID have not been established in patients with hypercholesterolemia who do not have HoFH, including those with heterozygous familial hypercholesterolemia (HeFH) ( 1 ).

The effect of JUXTAPID on cardiovascular morbidity and mortality has not been determined ( 1 ).

1.1Homozygous Familial Hypercholesterolemia JUXTAPID is indicated as an adjunct to a low-fat diet and other lipid-lowering treatments, including LDL apheresis where available, to reduce low-density lipoprotein cholesterol (LDL-C), total cholesterol (TC), apolipoprotein B (apo B), and non-high-density lipoprotein cholesterol (non-HDL-C) in patients with homozygous familial hypercholesterolemia (HoFH). Limitations of Use The safety and effectiveness of JUXTAPID have not been established in patients with hypercholesterolemia who do not have HoFH, including those with heterozygous familial hypercholesterolemia (HeFH).

The effect of JUXTAPID on cardiovascular morbidity and mortality has not been determined.

⏱️ Dosage and Administration ~3 min read ▾

2 DOSAGE AND ADMINISTRATION Before treatment, measure ALT, AST, alkaline phosphatase, and total bilirubin; obtain a negative pregnancy test in females of reproductive potential; and initiate a low-fat diet supplying <20% of energy from fat ( 2.1 ). Initiate treatment at 5 mg once daily. Titrate dose based on acceptable safety/tolerability: increase to 10 mg daily after at least 2 weeks; and then, at a minimum of 4-week intervals, to 20 mg, 40 mg, and up to the maximum recommended dose of 60 mg daily ( 2.1 ).

Due to reduced absorption of fat-soluble vitamins/fatty acids: Take daily vitamin E, linoleic acid, alpha-linolenic acid (ALA), eicosapentaenoic acid (EPA), and docosahexaenoic acid (DHA) supplements ( 2.1 , 5.4 ). Take once daily, whole, with water and without food, at least 2 hours after evening meal ( 2.2 ). Patients with end-stage renal disease on dialysis or with baseline mild hepatic impairment should not exceed 40 mg daily ( 2.5 , 2.6 ).

2.1Initiation and Maintenance of Therapy Before beginning treatment with JUXTAPID: Measure transaminases (ALT, AST), alkaline phosphatase, and total bilirubin [see Warnings and Precautions (5.1) ] ; Obtain a negative pregnancy test in females of reproductive potential prior to initiating treatment with JUXTAPID [see Contraindications (4) , Warnings and Precautions (5.3) , Use in Specific Populations (8.1 , 8.3) ] ; Initiate a low-fat diet supplying <20% of energy from fat [see Warnings and Precautions (5.5) ]. The recommended starting dosage of JUXTAPID is 5 mg once daily, and the dose should be escalated gradually based on acceptable safety and tolerability.

Transaminases should be measured prior to any increase in dose [see Warnings and Precautions (5.1) ] . The maintenance dosage of JUXTAPID should be individualized, taking into account patient characteristics such as goal of therapy and response to treatment, to a maximum of 60 mg daily as described in Table 1. Modify dosing for patients taking concomitant weak CYP3A4 inhibitors and for those with renal impairment or baseline hepatic impairment [see Dosage and Administration (2.3) , (2.5) , and (2.6) ] .

Monitor transaminases during treatment with JUXTAPID as described in Warnings and Precautions (5.1) , and reduce or withhold dosing for patients who develop transaminase values ≥3× the upper limit of normal (ULN) [see Dosage and Administration (2.4) ]. Table 1: Recommended Regimen for Titrating Dosage DOSAGE DURATION OF ADMINISTRATION BEFORE CONSIDERING INCREASE TO NEXT DOSAGE 5 mg daily At least 2 weeks 10 mg daily At least 4 weeks 20 mg daily At least 4 weeks 40 mg daily At least 4 weeks 60 mg daily Maximum recommended dosage To reduce the risk of developing a fat-soluble nutrient deficiency due to JUXTAPID's mechanism of action in the small intestine, patients treated with JUXTAPID should take daily supplements that contain 400 international units vitamin E and at least 200 mg linoleic acid, 210 mg alpha-linolenic acid (ALA), 110 mg eicosapentaenoic acid (EPA), and 80 mg docosahexaenoic acid (DHA) [see Warnings and Precautions (5.4) ].

2.2Administration JUXTAPID should be taken once daily with a glass of water, without food, at least 2 hours after the evening meal because administration with food may increase the risk of gastrointestinal adverse reactions [see Warnings and Precautions (5.5) ]. Patients should swallow JUXTAPID capsules whole. Capsules should not be opened, crushed, dissolved, or chewed.

2.3Dosing with Cytochrome P450 3A4 Inhibitors JUXTAPID is contraindicated with concomitant use of moderate and strong cytochrome P450 3A4 (CYP3A4) inhibitors [see Contraindications (4) and Drug Interactions (7.1) ]. The recommended maximum dosage of JUXTAPID is 30 mg daily with concomitant use of weak CYP3A4 inhibitors (such as alprazolam, amiodarone, amlodipine, atorvastatin, bicalutamide, cilostazol, cimetidine, cyclosporine, fluoxetine, fluvoxamine, ginkgo, goldenseal, isoniazid, lapatinib, nilotinib, pazopanib, ranitidin… [Excerpted — this section continues on DailyMed.]

💊 Dosage Forms and Strengths 74 words ▾

3 DOSAGE FORMS AND STRENGTHS 5 mg: Orange/orange hard gelatin capsule printed with black ink "A733" and "5 mg" 10 mg: Orange/white hard gelatin capsule printed with black ink "A733" and "10 mg" 20 mg: White/white hard gelatin capsule printed with black ink "A733" and "20 mg" 30 mg: Orange/yellow hard gelatin capsule printed with black ink "A733" and "30 mg" Capsules: 5 mg, 10 mg, 20 mg, and 30 mg ( 3 ).

⛔ Contraindications 128 words ▾

4 CONTRAINDICATIONS JUXTAPID is contraindicated in the following conditions: Pregnancy [see Warnings and Precautions (5.3) and Use in Specific Populations (8.1) ] . Concomitant administration of JUXTAPID with moderate or strong CYP3A4 inhibitors, as this can increase JUXTAPID exposure [see Warnings and Precautions (5.6) , Drug Interactions (7.1) , and Clinical Pharmacology (12.3) ]. Patients with moderate or severe hepatic impairment (based on Child-Pugh category B or C) and patients with active liver disease, including unexplained persistent elevations of serum transaminases [see Warnings and Precautions (5.1) and Use in Specific Populations (8.7) ] .

Pregnancy ( 4 ). Concomitant use with strong or moderate CYP3A4 inhibitors ( 4 ). Moderate or severe hepatic impairment or active liver disease including unexplained persistent abnormal liver function tests ( 4 ).

⚠️ Warnings and Cautions ~3 min read ▾

5 WARNINGS AND PRECAUTIONS Embryo-Fetal Toxicity: May cause fetal harm. Advise females of reproductive potential of the potential risk to the fetus and to use effective contraception. Discontinue JUXTAPID if pregnancy detected ( 5.3 , 8.1 , 8.3 ). Gastrointestinal adverse reactions occur in 93% of patients and could affect absorption of concomitant oral medications ( 5.5 ).

5.1Risk of Hepatotoxicity JUXTAPID can cause elevations in transaminases and hepatic steatosis, as described below [see Warnings and Precautions (5.2) ] . To what extent JUXTAPID-associated hepatic steatosis promotes the elevations in transaminases is unknown. Although cases of hepatic dysfunction (elevated transaminases with increase in bilirubin or INR) or hepatic failure have not been reported, there is concern that JUXTAPID could induce steatohepatitis, which can progress to cirrhosis over several years.

The clinical studies supporting the safety and efficacy of JUXTAPID in HoFH would have been unlikely to detect this adverse outcome given their size and duration [see Clinical Studies (14) ] . Elevation of Transaminases Elevations in transaminases (alanine aminotransferase [ALT] and/or aspartate aminotransferase [AST]) are associated with JUXTAPID. In the clinical trial, 10 (34%) of the 29 patients with HoFH had at least one elevation in ALT or AST ≥3× ULN, and 4 (14%) of the patients had at least one elevation in ALT or AST ≥5× ULN.

There were no concomitant or subsequent clinically meaningful elevations in bilirubin, INR, or alkaline phosphatase [see Adverse Reactions (6.1) ]. During the 78-week HoFH clinical trial, no patients discontinued prematurely because of elevated transaminases. Among the 19 patients who subsequently enrolled in the HoFH extension study, one discontinued because of increased transaminases that persisted despite several dose reductions, and one temporarily discontinued because of markedly elevated transaminases (ALT 24× ULN, AST 13× ULN) that had several possible causes, including a drug-drug interaction between JUXTAPID and the strong CYP3A4 inhibitor clarithromycin [see Drug Interactions (7.1) ].

Monitoring of Transaminases Before initiating JUXTAPID and during treatment, monitor transaminases as recommended in Table 3. Table 3: Recommendations for Monitoring Transaminases TIME RECOMMENDATIONS Before initiating treatment Measure ALT, AST, alkaline phosphatase, and total bilirubin. If abnormal, consider initiating JUXTAPID only after an appropriate work-up and the baseline abnormalities have been explained or resolved.

JUXTAPID is contraindicated in patients with moderate or severe hepatic impairment, or active liver disease, including unexplained persistent elevations of serum transaminases [see Contraindications (4) ] . During the first year Measure liver-related tests (ALT and AST, at a minimum) prior to each increase in dose or monthly, whichever occurs first. After the first year Measure liver-related tests (ALT and AST, at a minimum) at least every 3 months and before any increase in dose.

At any time during treatment If transaminases are abnormal, reduce or withhold dosing of JUXTAPID and monitor as recommended [see Dosage and Administration (2.4) ]. Discontinue JUXTAPID for persistent or clinically significant elevations. If transaminase elevations are accompanied by clinical symptoms of liver injury (such as nausea, vomiting, abdominal pain, fever, jaundice, lethargy, flu-like symptoms), increases in bilirubin ≥2× ULN, or active liver disease, discontinue treatment with JUXTAPID and identify the probable cause.

Hepatic Steatosis JUXTAPID increases hepatic fat, with or without concomitant increases in transaminases. Hepatic steatosis is a risk factor for progressive liver disease, including steatohepatitis and cirrhosis. The long-term consequences of hepatic steatosis associated with JUXTAPID treatment are unknown.

During the HoFH clinical trial, the median absolute increase in hepatic fat was 6% after both 26 weeks a… [Excerpted — this section continues on DailyMed.]

🤒 Adverse Reactions ~3 min read ▾

6 ADVERSE REACTIONS The following important adverse reactions have been observed and are discussed in detail in other sections of the label: Risk of hepatotoxicity [see Warnings and Precautions (5.1) ] Reduced absorption of fat-soluble vitamins, and serum fatty acids [see Warnings and Precautions (5.4) ] Gastrointestinal adverse reactions [see Warnings and Precautions (5.5) ] Most common adverse reactions (incidence ≥28%) are diarrhea, nausea, vomiting, dyspepsia, and abdominal pain ( 6.1 ). To report SUSPECTED ADVERSE REACTIONS, contact Amryt Pharmaceuticals DAC at 1-855-303-2347 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

6.1Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. One single-arm, open-label, 78-week trial has been conducted in 29 patients with HoFH, 23 of whom completed at least one year of treatment. The initial dosage of JUXTAPID was 5 mg daily, with titration up to 60 mg daily during an 18-week period based on safety and tolerability.

In this trial, the mean age was 30.7 years (range, 18 to 55 years), 16 (55%) patients were men, 25 (86%) patients were Caucasian, 2 (7%) were Asian, 1 (3%) was African American, and 1 (3%) was multi-racial [see Clinical Studies (14) ] . Five (17%) of the 29 patients with HoFH that participated in the clinical trial discontinued treatment due to an adverse reaction. The adverse reactions that contributed to treatment discontinuations included diarrhea (2 patients; 7%) and abdominal pain, nausea, gastroenteritis, weight loss, headache, and difficulty controlling INR on warfarin (1 patient each; 3%).

The most common adverse reactions were gastrointestinal, reported by 27 (93%) of 29 patients. Adverse reactions reported by ≥8 (28%) patients in the HoFH clinical trial included diarrhea, nausea, vomiting, dyspepsia, and abdominal pain. Other common adverse reactions, reported by 5 to 7 (17-24%) patients, included weight loss, abdominal discomfort, abdominal distension, constipation, flatulence, increased ALT, chest pain, influenza, nasopharyngitis, and fatigue.

The adverse reactions reported in at least 10% of patients during the HoFH clinical trial are presented in Table 4. Table 4: Adverse Reactions Reported in ≥10% of Patients in the Clinical Trial in HoFH ADVERSE REACTION N (%) Gastrointestinal Disorders Diarrhea 23 (79) Nausea 19 (65) Dyspepsia 11 (38) Vomiting 10 (34) Abdominal pain 10 (34) Abdominal discomfort 6 (21) Abdominal distension 6 (21) Constipation 6 (21) Flatulence 6 (21) Gastroesophageal reflux disease 3 (10) Defecation urgency 3 (10) Rectal tenesmus 3 (10) Infections Influenza 6 (21) Nasopharyngitis 5 (17) Gastroenteritis 4 (14) Investigations Decreased weight 7 (24) Increased ALT 5 (17) General Disorders Chest pain 7 (24) Fatigue 5 (17) Fever 3 (10) Musculoskeletal Disorders Back pain 4 (14) Nervous System Disorders Headache 3 (10) Dizziness 3 (10) Respiratory Disorders Pharyngolaryngeal pain 4 (14) Nasal congestion 3 (10) Cardiac Disorders Angina pectoris 3 (10) Palpitations 3 (10) Adverse reactions of severe intensity were reported by 8 (28%) of 29 patients, with the most common being diarrhea (4 patients, 14%), vomiting (3 patients, 10%), increased ALT or hepatotoxicity (3 patients, 10%), and abdominal pain, distension, and/or discomfort (2 patients, 7%).

Transaminase Elevations During the HoFH clinical trial, 10 (34%) of 29 patients had at least one elevation in ALT and/or AST ≥3× ULN (see Table 5 ). No clinically meaningful elevations in total bilirubin or alkaline phosphatase were observed. Transaminases typically fell within one to four weeks of reducing the dose or withholding JUXTAPID.

Table 5: Patient Incidence of Transaminase Elevations During the HoFH Clinical Trial N (%) Upper limits of normal (ULN) ranged from… [Excerpted — this section continues on DailyMed.]

🔄 Drug Interactions ~2 min read ▾

7 DRUG INTERACTIONS CYP3A4 inhibitors increase exposure to lomitapide. Strong and moderate CYP3A4 inhibitors are contraindicated with JUXTAPID. Patients must avoid grapefruit juice ( 4 , 5.6 , 7.1 ).

When administered with weak CYP3A4 inhibitors, the dose of JUXTAPID should be decreased by half. The dosage of JUXTAPID may then be up-titrated to a maximum recommended dosage of 30 mg daily ( 2.3 , 5.6 , 7.2 ). Warfarin: Lomitapide increases plasma concentrations of warfarin.

Monitor international normalized ratio (INR) regularly, especially with JUXTAPID dose adjustment ( 5.8 , 7.3 ). Simvastatin and lovastatin exposure increase with JUXTAPID. Limit dose when co-administered with JUXTAPID due to myopathy risk ( 5.7 , 7.4 ).

P-glycoprotein (P-gp) Substrates: Consider dose reduction of P-gp substrate because of possible increased absorption with JUXTAPID ( 7.5 ). Bile Acid Sequestrants: Separate JUXTAPID dosing by at least 4 hours ( 7.6 ).

7.1Moderate and Strong CYP3A4 Inhibitors A strong CYP3A4 inhibitor has been shown to increase lomitapide exposure approximately 27-fold [see Clinical Pharmacology (12.3) ] . Concomitant use of strong CYP3A4 inhibitors (such as boceprevir, clarithromycin, conivaptan, indinavir, itraconazole, ketoconazole, lopinavir/ritonavir, nefazodone, nelfinavir, posaconazole, ritonavir, saquinavir, telaprevir, telithromycin, tipranavir/ritonavir, voriconazole) with lomitapide is contraindicated. Concomitant use of moderate CYP3A4 inhibitors (such as amprenavir, aprepitant, atazanavir, ciprofloxacin, crizotinib, darunavir/ritonavir, diltiazem, erythromycin, fluconazole, fosamprenavir, imatinib, verapamil) has not been studied, but concomitant use with lomitapide is contraindicated since lomitapide exposure will likely increase significantly in the presence of these inhibitors.

Patients must avoid grapefruit juice while taking JUXTAPID [see Contraindications (4) , Warnings and Precautions (5.6) , and Clinical Pharmacology (12.3) ] .

7.2Weak CYP3A4 Inhibitors Weak CYP3A4 inhibitors (such as alprazolam, amiodarone, amlodipine, atorvastatin, bicalutamide, cilostazol, cimetidine, cyclosporine, fluoxetine, fluvoxamine, ginkgo, goldenseal, isoniazid, lapatinib, nilotinib, pazopanib, ranitidine, ranolazine, ticagrelor, zileuton) can increase lomitapide exposure approximately 2-fold [see Clinical Pharmacology (12.3) ] . When administered with weak CYP3A4 inhibitors, the dose of JUXTAPID should be decreased by half. Careful titration of JUXTAPID may then be considered based on LDL-C response and safety/tolerability to a maximum recommended dosage of 30 mg daily except when coadministered with oral contraceptives, in which case the maximum recommended lomitapide dosage is 40 mg daily [see Dosage and Administration (2.3) , Warnings and Precautions (5.6) , and Clinical Pharmacology (12.3) ] .

7.3Warfarin Lomitapide increases plasma concentrations of both R(+)-warfarin and S(-)-warfarin by approximately 30% and increased the INR 22%. Patients taking warfarin should undergo regular monitoring of INR, particularly after any changes in lomitapide dosage. The dose of warfarin should be adjusted as clinically indicated [see Warnings and Precautions (5.8) ] .

7.4Simvastatin and Lovastatin The risk of myopathy, including rhabdomyolysis, with simvastatin and lovastatin monotherapy is dose related. Lomitapide approximately doubles the exposure of simvastatin; therefore, the recommended dose of simvastatin should be reduced by 50% when initiating JUXTAPID [see Clinical Pharmacology (12.3) ] . While taking JUXTAPID, limit simvastatin dosage to 20 mg daily (or 40 mg daily for patients who have previously tolerated simvastatin 80 mg daily for at least one year without evidence of muscle toxicity).

Refer to the simvastatin prescribing information for simvastatin dosing recommendations. Interaction between lovastatin and lomitapide has not been studied. However, the metabolizing enzymes and transporters responsible for the disposition… [Excerpted — this section continues on DailyMed.]

👥 Use in Specific Populations ~3 min read ▾

8 USE IN SPECIFIC POPULATIONS Lactation: Breastfeeding not recommended ( 8.2 ). Pediatric Patients: Safety and effectiveness not established ( 8.4 ).

8.1Pregnancy Pregnancy Exposure Registry There is a pregnancy exposure registry that monitors pregnancy outcomes in women exposed to JUXTAPID during pregnancy. For additional information visit www.JUXTAPID.com or call the Global Lomitapide Pregnancy Exposure Registry (PER) at 1-877-902-4099. Healthcare professionals are encouraged to call the PER at 1-877-902-4099 to enroll patients who become pregnant during JUXTAPID treatment.

Risk Summary Based on findings from animal studies, JUXTAPID use is contraindicated in pregnancy since it may cause fetal harm [see Contraindications (4) , Warnings and Precautions (5.3) ]. Available human data are insufficient to draw conclusions about any drug-associated risks for major birth defects, miscarriage, or adverse maternal or fetal outcomes. However, in animal reproduction studies, lomitapide was teratogenic in rats at clinically relevant exposures and in ferrets at exposures estimated to be less than human therapeutic exposure at 60 mg when administered during organogenesis, based on AUC comparisons.

Embryo-fetal lethality was observed in rabbits at 6-times the maximum recommended human dose (MRHD) of 60 mg based on body surface area. If pregnancy is detected, discontinue JUXTAPID. The estimated background risk of major birth defects and miscarriage for the indicated population is unknown.

In the U.S. general population, the estimated background risks of major birth defects and miscarriage in clinically recognized pregnancies is 2-4% and 15-20%, respectively. Data Animal Data Oral gavage doses of 0.04, 0.4, or 4 mg/kg/day lomitapide given to pregnant rats from gestation day 6 through organogenesis were associated with fetal malformations at ≥2-times human exposure at the MRHD (60 mg) based on plasma AUC comparisons. Fetal malformations included umbilical hernia, gastroschisis, imperforate anus, alterations in heart shape and size, limb malrotations, skeletal malformations of the tail, and delayed ossification of cranial, vertebral and pelvic bones.

Oral gavage doses of 1.6, 4, 10, or 25 mg/kg/day lomitapide given to pregnant ferrets from gestation day 12 through organogenesis were associated with both maternal toxicity and fetal malformations at exposures that ranged from less than the human exposure at the MRHD to 5-times the human exposure at the MRHD. Fetal malformations included umbilical hernia, medially rotated or short limbs, absent or fused digits on paws, cleft palate, open eye lids, low-set ears, and kinked tail. Oral gavage doses of 0.1, 1, or 10 mg/kg/day lomitapide given to pregnant rabbits from gestation day 6 through organogenesis were not associated with adverse effects at systemic exposures up to 3-times the MRHD of 60 mg based on body surface area comparison.

Treatment at doses of ≥20 mg/kg/day, ≥6-times the MRHD, resulted in embryo-fetal lethality. Pregnant female rats given oral gavage doses of 0.1, 0.3, or 1 mg/kg/day lomitapide from gestation day 7 through termination of nursing on lactation day 20 were associated with malformations at systemic exposures equivalent to human exposure at the MRHD of 60 mg based on AUC. Increased pup mortality occurred at 4-times the MRHD.

8.2Lactation Risk Summary There are no data on the presence of lomitapide in human or animal milk, effects on the breastfed infant or on milk production. Because of the potential for serious adverse reactions, including hepatotoxicity, advise patients that breastfeeding is not recommended during treatment with JUXTAPID.

8.3Females and Males of Reproductive Potential Pregnancy Testing Females of reproductive potential should have a negative pregnancy test before starting JUXTAPID. Contraception Based on animal studies, JUXTAPID may cause fetal harm when administered to pregnant women [see Use in Specific Populations (8.1) ]. Advise females of reproduct… [Excerpted — this section continues on DailyMed.]

🤰 Pregnancy ~2 min read ▾

8.1Pregnancy Pregnancy Exposure Registry There is a pregnancy exposure registry that monitors pregnancy outcomes in women exposed to JUXTAPID during pregnancy. For additional information visit www.JUXTAPID.com or call the Global Lomitapide Pregnancy Exposure Registry (PER) at 1-877-902-4099. Healthcare professionals are encouraged to call the PER at 1-877-902-4099 to enroll patients who become pregnant during JUXTAPID treatment.

Risk Summary Based on findings from animal studies, JUXTAPID use is contraindicated in pregnancy since it may cause fetal harm [see Contraindications (4) , Warnings and Precautions (5.3) ]. Available human data are insufficient to draw conclusions about any drug-associated risks for major birth defects, miscarriage, or adverse maternal or fetal outcomes. However, in animal reproduction studies, lomitapide was teratogenic in rats at clinically relevant exposures and in ferrets at exposures estimated to be less than human therapeutic exposure at 60 mg when administered during organogenesis, based on AUC comparisons.

Embryo-fetal lethality was observed in rabbits at 6-times the maximum recommended human dose (MRHD) of 60 mg based on body surface area. If pregnancy is detected, discontinue JUXTAPID. The estimated background risk of major birth defects and miscarriage for the indicated population is unknown.

In the U.S. general population, the estimated background risks of major birth defects and miscarriage in clinically recognized pregnancies is 2-4% and 15-20%, respectively. Data Animal Data Oral gavage doses of 0.04, 0.4, or 4 mg/kg/day lomitapide given to pregnant rats from gestation day 6 through organogenesis were associated with fetal malformations at ≥2-times human exposure at the MRHD (60 mg) based on plasma AUC comparisons. Fetal malformations included umbilical hernia, gastroschisis, imperforate anus, alterations in heart shape and size, limb malrotations, skeletal malformations of the tail, and delayed ossification of cranial, vertebral and pelvic bones.

Oral gavage doses of 1.6, 4, 10, or 25 mg/kg/day lomitapide given to pregnant ferrets from gestation day 12 through organogenesis were associated with both maternal toxicity and fetal malformations at exposures that ranged from less than the human exposure at the MRHD to 5-times the human exposure at the MRHD. Fetal malformations included umbilical hernia, medially rotated or short limbs, absent or fused digits on paws, cleft palate, open eye lids, low-set ears, and kinked tail. Oral gavage doses of 0.1, 1, or 10 mg/kg/day lomitapide given to pregnant rabbits from gestation day 6 through organogenesis were not associated with adverse effects at systemic exposures up to 3-times the MRHD of 60 mg based on body surface area comparison.

Treatment at doses of ≥20 mg/kg/day, ≥6-times the MRHD, resulted in embryo-fetal lethality. Pregnant female rats given oral gavage doses of 0.1, 0.3, or 1 mg/kg/day lomitapide from gestation day 7 through termination of nursing on lactation day 20 were associated with malformations at systemic exposures equivalent to human exposure at the MRHD of 60 mg based on AUC. Increased pup mortality occurred at 4-times the MRHD.

🧒 Pediatric Use 13 words ▾

8.4Pediatric Use Safety and effectiveness have not been established in pediatric patients.

🧓 Geriatric Use 73 words ▾

8.5Geriatric Use Clinical studies of JUXTAPID did not include sufficient numbers of patients aged 65 years and over to determine whether they respond differently from younger patients. Other reported clinical experience has not identified differences in responses between the elderly and younger patients. In general, dosing for an elderly patient should be cautious, reflecting the greater frequency of decreased hepatic, renal, or cardiac function, and of concomitant disease or other drug therapy.

🆘 Overdosage 49 words ▾

10 OVERDOSAGE There is no specific treatment in the event of overdose of JUXTAPID. In the event of overdose, the patient should be treated symptomatically and supportive measures instituted as required. Liver-related tests should be monitored. Hemodialysis is unlikely to be beneficial given that lomitapide is highly protein bound.

🧬 Clinical Pharmacology ~3 min read ▾

12 CLINICAL PHARMACOLOGY

12.1Mechanism of Action JUXTAPID directly binds and inhibits microsomal triglyceride transfer protein (MTP), which resides in the lumen of the endoplasmic reticulum, thereby preventing the assembly of apo B-containing lipoproteins in enterocytes and hepatocytes. This inhibits the synthesis of chylomicrons and VLDL. The inhibition of the synthesis of VLDL leads to reduced levels of plasma LDL-C.

12.2Pharmacodynamics Effects on QT Interval At a concentration 23 times the C max of the maximum recommended dose, lomitapide does not prolong QTc to any clinically relevant extent.

12.3Pharmacokinetics Absorption Upon oral administration of a single 60-mg dose of JUXTAPID, the lomitapide t max is around 6 hours in healthy volunteers. The absolute bioavailability of lomitapide is approximately 7%. Lomitapide pharmacokinetics is approximately dose-proportional for oral single doses from 10-100 mg.

Distribution The mean lomitapide volume of distribution at steady state is 985-1292 liters. Lomitapide is 99.8% plasma-protein bound. Metabolism Lomitapide is metabolized extensively by the liver.

The metabolic pathways include oxidation, oxidative N-dealkylation, glucuronide conjugation, and piperidine ring opening. Cytochrome P450 (CYP) 3A4 metabolizes lomitapide to its major metabolites, M1 and M3, as detected in plasma. The oxidative N-dealkylation pathway breaks the lomitapide molecule into M1 and M3.

M1 is the moiety that retains the piperidine ring, whereas M3 retains the rest of the lomitapide molecule in vitro . CYPs 1A2, 2B6, 2C8, and 2C19 may metabolize lomitapide to a small extent to M1. M1 and M3 do not inhibit activity of microsomal triglyceride transfer protein in vitro .

Excretion In a mass-balance study, a mean of 59.5% and 33.4% of the dose was excreted in the urine and feces, respectively. In another mass-balance study, a mean of 52.9% and 35.1% of the dose was excreted in the urine and feces, respectively. Lomitapide was not detectable in urine samples.

M1 is the major urinary metabolite. Lomitapide is the major component in the feces. The mean lomitapide terminal half-life is 39.7 hours.

Specific Populations Hepatic Impairment A single-dose, open-label study was conducted to evaluate the pharmacokinetics of 60 mg lomitapide in healthy volunteers with normal hepatic function compared with patients with mild (Child-Pugh A) and moderate (Child-Pugh B) hepatic impairment. In patients with moderate hepatic impairment, lomitapide AUC and C max were 164% and 361% higher, respectively, compared with healthy volunteers. In patients with mild hepatic impairment, lomitapide AUC and C max were 47% and 4% higher, respectively, compared with healthy volunteers.

Lomitapide has not been studied in patients with severe hepatic impairment (Child-Pugh score 10-15) [see Dosage and Administration (2.6) , Contraindications (4) , Warnings and Precautions (5.1) , and Use in Specific Populations (8.7) ] . Renal Impairment A single-dose, open-label study was conducted to evaluate the pharmacokinetics of 60 mg lomitapide in patients with end-stage renal disease receiving hemodialysis compared with healthy volunteers with normal renal function. Healthy volunteers had estimated creatinine clearance >80 mL/min by the Cockcroft-Gault equation.

Compared with healthy volunteers, lomitapide AUC 0-inf and C max were 40% and 50% higher, respectively, in patients with end-stage renal disease receiving hemodialysis. Effects of mild, moderate, and severe renal impairment as well as end-stage renal disease not yet on dialysis on lomitapide exposure have not been studied [see Dosage and Administration (2.5) and Use in Specific Populations (8.6) ] . Drug Interactions [see Dosage and Administration (2.3) , Contraindications (4) , Warnings and Precautions (5.6) , (5.7) , (5.8) , and Drug Interactions (7) ].

In vitro Assessment of Drug Interactions Lomitapide does not induce CYPs 1A2, 3A4, or 2B6. Lomitapide inhibits CYP3A4. Lomitapide does… [Excerpted — this section continues on DailyMed.]

🧬 Mechanism of Action 57 words ▾

12.1Mechanism of Action JUXTAPID directly binds and inhibits microsomal triglyceride transfer protein (MTP), which resides in the lumen of the endoplasmic reticulum, thereby preventing the assembly of apo B-containing lipoproteins in enterocytes and hepatocytes. This inhibits the synthesis of chylomicrons and VLDL. The inhibition of the synthesis of VLDL leads to reduced levels of plasma LDL-C.

📦 How Supplied / Storage and Handling 141 words ▾

16 HOW SUPPLIED / STORAGE AND HANDLING 5 mg capsules: Orange/orange hard gelatin capsule printed with black ink "A733" and "5 mg" Bottles of 28 NDC 76431-105-01 10 mg capsules: Orange/white hard gelatin capsule printed with black ink "A733" and "10 mg" Bottles of 28 NDC 76431-110-01 20 mg capsules: White/white hard gelatin capsule printed with black ink "A733" and "20 mg" Bottles of 28 NDC 76431-120-01 30 mg capsules: Orange/yellow hard gelatin capsule printed with black ink "A733" and "30 mg" Bottles of 28 NDC 76431-130-01 Storage: Store at 20°C to 25°C (68°F to 77°F); excursions permitted between 15°C and 30°C (between 59°F and 86°F).

Brief exposure to temperatures up to 40°C (104°F) may be tolerated provided the mean kinetic temperature does not exceed 25°C (77°F); however, such exposure should be minimized. Keep container tightly closed and protect from moisture.

📦 Storage and Handling 54 words ▾

Storage: Store at 20°C to 25°C (68°F to 77°F); excursions permitted between 15°C and 30°C (between 59°F and 86°F). Brief exposure to temperatures up to 40°C (104°F) may be tolerated provided the mean kinetic temperature does not exceed 25°C (77°F); however, such exposure should be minimized. Keep container tightly closed and protect from moisture.

📋 Description 195 words ▾

11 DESCRIPTION JUXTAPID capsules contain lomitapide mesylate, a synthetic lipid-lowering agent for oral administration. The chemical name of lomitapide mesylate is N-(2,2,2-trifluoroethyl)-9-[4-[4-[[[4'-(trifluoromethyl)[1,1'-biphenyl]-2-yl]carbonyl]amino]-1-piperidinyl]butyl]-9 H -fluorene-9-carboxamide, methanesulfonate salt. Its structural formula is: The empirical formula for lomitapide mesylate is C 39 H 37 F 6 N 3 O 2 ∙ CH 4 O 3 S and its molecular weight is 789.8.

Lomitapide mesylate is a white to off-white powder that is slightly soluble in aqueous solutions of pH 2 to 5. Lomitapide mesylate is freely soluble in acetone, ethanol, and methanol; soluble in 2-butanol, methylene chloride, and acetonitrile; sparingly soluble in 1-octanol and 2-propanol; slightly soluble in ethyl acetate; and insoluble in heptane. Each JUXTAPID capsule contains lomitapide mesylate equivalent to 5, 10, 20, or 30 mg lomitapide free base and the following inactive ingredients: pregelatinized starch, sodium starch glycolate, microcrystalline cellulose, lactose monohydrate, silicon dioxide and magnesium stearate.

The capsule shells of all strengths contain gelatin and titanium dioxide; the 5 mg, 10 mg and 30 mg capsules also contain red iron oxide; and the 30 mg capsules also contain yellow iron oxide. The imprinting ink contains shellac, black iron oxide, and propylene glycol. Chemical Structure

💬 Information for Patients ~3 min read ▾

17 PATIENT COUNSELING INFORMATION See FDA-approved labeling (Medication Guide) Patients should be informed that a registry for patients taking JUXTAPID has been established in order to monitor and evaluate the long-term effects of JUXTAPID. Patients are encouraged to participate in the registry and should be informed that their participation is voluntary. For information regarding the registry program visit www.JUXTAPID.com or call 1-877-902-4099.

Advise patients of the following: Risk of Hepatotoxicity [see Warnings and Precautions (5.1) ] JUXTAPID can cause both elevations in transaminases and hepatic steatosis. Discuss with the patient the importance of monitoring of liver-related tests before taking JUXTAPID, prior to each dose escalation, and periodically thereafter. Patients should be advised of the potential for increased risk of liver injury if alcohol is consumed while taking JUXTAPID.

It is recommended that patients taking JUXTAPID limit consumption to not more than one alcoholic drink per day. JUXTAPID is commonly associated with nausea, vomiting, and abdominal pain. Advise patients to promptly report these symptoms if they increase in severity, persist, or change in the character, as they might reflect liver injury.

Patients should also report any other symptoms of possible liver injury, including fever, jaundice, lethargy, or flu-like symptoms. JUXTAPID REMS Program [see Warnings and Precautions (5.2) ] JUXTAPID is only available through a restricted program called JUXTAPID REMS Program and therefore, JUXTAPID is only available from certified pharmacies that are enrolled in the program. Embryofetal Toxicity [see Warnings and Precautions (5.3) , Drug Interactions (7.2) , Use in Specific Populations (8.1 , 8.3) ] JUXTAPID is contraindicated in pregnancy since it may cause fetal harm.

Advise females who become pregnant to discontinue JUXTAPID and inform their healthcare provider of a known or suspected pregnancy. Advise females of reproductive potential to use effective contraception during treatment with JUXTAPID and for two weeks after the final dose. Advise patients who are taking oral contraceptives and experience vomiting or diarrhea while taking JUXTAPID to use an effective alternative contraceptive method until 7 days after resolution of symptoms .

Lactation [see Use in Specific Populations (8.2) ] Advise females not to breastfeed during treatment with JUXTAPID . Dietary Supplements [see Warnings and Precautions (5.4) ] Discuss with the patient the importance of taking daily supplements that contain 400 international units vitamin E and at least 200 mg linoleic acid, 210 mg alpha-linolenic acid (ALA), 110 mg eicosapentaenoic acid (EPA), and 80 mg docosahexaenoic acid (DHA). Gastrointestinal Adverse Reactions [see Warnings and Precautions (5.5) ] Inform the patient that gastrointestinal adverse reactions are common with JUXTAPID.

These include, but are not limited to, diarrhea, nausea/vomiting, abdominal pain/discomfort, flatulence, and constipation. Strict adherence to a low-fat diet (<20% of total calories from fat) may reduce these reactions. Instruct the patient to stop JUXTAPID and contact their healthcare provider if severe diarrhea occurs or if they experience symptoms of volume depletion such as lightheadedness, decreased urine output, or tiredness.

Tell the patient that taking JUXTAPID with food may adversely impact gastrointestinal tolerability; therefore, they should take JUXTAPID at least 2 hours after the evening meal, swallowing each capsule whole. Absorption of oral medications may be affected in patients who develop diarrhea or vomiting. For example, hormone absorption from oral contraceptives may be incomplete, warranting the use of additional contraceptive methods.

Patients who develop these symptoms should seek advice from their healthcare provider. Drug Interactions [see Warnings and Precautions (5.6) and Drug Interactions (7) ] Tell the patient to omit grapefruit juice from his/her diet whi… [Excerpted — this section continues on DailyMed.]

💬 Medication Guide ~3 min read ▾

Medication Guide JUXTAPID ® (JUKS-tuh-pid) (lomitapide) capsules What is the most important information I should know about JUXTAPID? JUXTAPID is available only through certified pharmacies that are enrolled in the JUXTAPID REMS Program. Your doctor must be enrolled in the program in order for you to be prescribed JUXTAPID.

There is a registry that collects information about the effects of taking JUXTAPID over time. Ask your doctor for more information about this registry or visit www.JUXTAPID.com or call 1-877-902-4099. JUXTAPID may cause serious side effects including: 1.

Liver problems. JUXTAPID can cause liver problems such as increased liver enzymes or increased fat in the liver. Your doctor should do blood tests to check your liver before you start JUXTAPID, if your dose is increased, and while you take JUXTAPID.

If your tests show some liver problems, your doctor may adjust your dose of JUXTAPID or stop it altogether. Tell your doctor if you have had liver problems, including liver problems while taking other medicines. JUXTAPID may cause nausea, vomiting and stomach pain, especially if you do not eat a low-fat diet.

These side effects can also be symptoms of liver problems. Tell your doctor right away if you have any of these symptoms of liver problems while taking JUXTAPID: Nausea, vomiting, or stomach pain that gets worse, does not go away, or changes fever flu-like symptoms yellowing of your eyes or skin you are more tired than usual Drinking alcohol may increase your chance of having liver problems or make your liver problems worse. You should not have more than 1 alcoholic drink each day while taking JUXTAPID.

2. Harm to your unborn baby. JUXTAPID may cause harm to your unborn baby.

If you are pregnant, think you may be pregnant, or are planning to become pregnant, do not take JUXTAPID. If you are a female who can get pregnant, you should have a pregnancy test before you start taking JUXTAPID. Your pregnancy test must be negative for you to get JUXTAPID.

Do not have sex while taking JUXTAPID unless you are using effective birth control. Talk with your doctor or nurse to find the best method of birth control for you. Birth control pills may not work as well if you have diarrhea or vomiting.

If you start taking birth control pills while you are taking JUXTAPID, tell your doctor. Your doctor might need to change your dose of JUXTAPID. If you become pregnant while taking JUXTAPID, stop taking JUXTAPID and call your doctor right away.

Pregnancy Exposure Registry: There is a pregnancy exposure registry that monitors outcomes in women exposed to JUXTAPID during pregnancy. If you become pregnant while taking JUXTAPID, call 1-877-902-4099 or visit www.JUXTAPID.com for more information about the JUXTAPID pregnancy exposure registry. What is JUXTAPID?

JUXTAPID is a prescription medicine used along with diet and other lipid-lowering treatments, including low-density lipoprotein (LDL) apheresis where available, in people with homozygous familial hypercholesterolemia (HoFH) to reduce: LDL ("bad") cholesterol total cholesterol a protein that carries "bad" cholesterol in the blood (apolipoprotein B) non-high-density lipoprotein cholesterol (non-HDL-C) It is not known if JUXTAPID can decrease problems from high cholesterol, such as heart attack, stroke, death or other health problems.

It is not known if JUXTAPID is safe and effective in people with high cholesterol who do not have HoFH, including those with heterozygous familial hypercholesterolemia (HeFH). JUXTAPID should not be taken with certain medicines or food. You should not drink grapefruit juice.

Ask your doctor or pharmacist to make sure you can take JUXTAPID with your other medicines, and tell your doctor about any new medicines you might take even for only a short time. It is not known if JUXTAPID is safe and effective in people with kidney problems including people with end-stage kidney disease who are not on dialysis. It is not known if JUXTAPID is safe and effective… [Excerpted — this section continues on DailyMed.]

🧬 Pharmacokinetics ~3 min read ▾

12.3Pharmacokinetics Absorption Upon oral administration of a single 60-mg dose of JUXTAPID, the lomitapide t max is around 6 hours in healthy volunteers. The absolute bioavailability of lomitapide is approximately 7%. Lomitapide pharmacokinetics is approximately dose-proportional for oral single doses from 10-100 mg.

Distribution The mean lomitapide volume of distribution at steady state is 985-1292 liters. Lomitapide is 99.8% plasma-protein bound. Metabolism Lomitapide is metabolized extensively by the liver.

The metabolic pathways include oxidation, oxidative N-dealkylation, glucuronide conjugation, and piperidine ring opening. Cytochrome P450 (CYP) 3A4 metabolizes lomitapide to its major metabolites, M1 and M3, as detected in plasma. The oxidative N-dealkylation pathway breaks the lomitapide molecule into M1 and M3.

M1 is the moiety that retains the piperidine ring, whereas M3 retains the rest of the lomitapide molecule in vitro . CYPs 1A2, 2B6, 2C8, and 2C19 may metabolize lomitapide to a small extent to M1. M1 and M3 do not inhibit activity of microsomal triglyceride transfer protein in vitro .

Excretion In a mass-balance study, a mean of 59.5% and 33.4% of the dose was excreted in the urine and feces, respectively. In another mass-balance study, a mean of 52.9% and 35.1% of the dose was excreted in the urine and feces, respectively. Lomitapide was not detectable in urine samples.

M1 is the major urinary metabolite. Lomitapide is the major component in the feces. The mean lomitapide terminal half-life is 39.7 hours.

Specific Populations Hepatic Impairment A single-dose, open-label study was conducted to evaluate the pharmacokinetics of 60 mg lomitapide in healthy volunteers with normal hepatic function compared with patients with mild (Child-Pugh A) and moderate (Child-Pugh B) hepatic impairment. In patients with moderate hepatic impairment, lomitapide AUC and C max were 164% and 361% higher, respectively, compared with healthy volunteers. In patients with mild hepatic impairment, lomitapide AUC and C max were 47% and 4% higher, respectively, compared with healthy volunteers.

Lomitapide has not been studied in patients with severe hepatic impairment (Child-Pugh score 10-15) [see Dosage and Administration (2.6) , Contraindications (4) , Warnings and Precautions (5.1) , and Use in Specific Populations (8.7) ] . Renal Impairment A single-dose, open-label study was conducted to evaluate the pharmacokinetics of 60 mg lomitapide in patients with end-stage renal disease receiving hemodialysis compared with healthy volunteers with normal renal function. Healthy volunteers had estimated creatinine clearance >80 mL/min by the Cockcroft-Gault equation.

Compared with healthy volunteers, lomitapide AUC 0-inf and C max were 40% and 50% higher, respectively, in patients with end-stage renal disease receiving hemodialysis. Effects of mild, moderate, and severe renal impairment as well as end-stage renal disease not yet on dialysis on lomitapide exposure have not been studied [see Dosage and Administration (2.5) and Use in Specific Populations (8.6) ] . Drug Interactions [see Dosage and Administration (2.3) , Contraindications (4) , Warnings and Precautions (5.6) , (5.7) , (5.8) , and Drug Interactions (7) ].

In vitro Assessment of Drug Interactions Lomitapide does not induce CYPs 1A2, 3A4, or 2B6. Lomitapide inhibits CYP3A4. Lomitapide does not inhibit CYPs 1A2, 2B6, 2C9, 2C19, 2D6, or 2E1.

M1 and M3 do not induce CYPs 1A2, 3A4, or 2B6. M1 and M3 do not inhibit CYPs 1A2, 2A6, 2B6, 2C8, 2C9, 2C19, 2D6, 2E1, or 3A4. Lomitapide is not a P-gp substrate.

Lomitapide inhibits P-gp but does not inhibit breast cancer resistance protein (BCRP). Effects of other Drugs on Lomitapide Table 6 summarizes the effect of coadministered drugs on lomitapide AUC and C max . Table 6: Effect of Coadministered Drugs on Lomitapide Systemic Exposure COADMINISTERED DRUG DOSING OF COADMINISTERED DRUG DOSING OF LOMITAPIDE RATIO OF LOMITAPIDE EXPOSURE WITH/WITHOUT… [Excerpted — this section continues on DailyMed.]

🧬 Pharmacodynamics 29 words ▾

12.2Pharmacodynamics Effects on QT Interval At a concentration 23 times the C max of the maximum recommended dose, lomitapide does not prolong QTc to any clinically relevant extent.

🔬 Clinical Studies ~3 min read ▾

14 CLINICAL STUDIES The safety and effectiveness of JUXTAPID as an adjunct to a low-fat diet and other lipid-lowering treatments, including LDL apheresis where available, were evaluated in a multinational, single-arm, open-label, 78-week trial involving 29 adults with HoFH. A diagnosis of HoFH was defined by the presence of at least one of the following clinical criteria: (1) documented functional mutation(s) in both LDL receptor alleles or alleles known to affect LDL receptor functionality, or (2) skin fibroblast LDL receptor activity <20% normal, or (3) untreated TC >500 mg/dL and TG <300 mg/dL and both parents with documented untreated TC >250 mg/dL.

Among the 29 patients enrolled, the mean age was 30.7 years (range, 18 to 55 years), 16 (55%) were men, and the majority (86%) were Caucasian. The mean body mass index (BMI) was 25.8 kg/m 2 , with four patients meeting BMI criteria for obesity; one patient had type 2 diabetes. Concomitant lipid-lowering treatments at baseline included one or more of the following: statins (93%), ezetimibe (76%), nicotinic acid (10%), bile acid sequestrant (3%), and fibrate (3%); 18 (62%) were receiving apheresis.

After a six-week run-in period to stabilize lipid-lowering treatments, including the establishment of an LDL apheresis schedule if applicable, JUXTAPID was initiated at 5 mg daily and titrated to daily doses of 10 mg, 20 mg, 40 mg, and 60 mg at weeks 2, 6, 10, and 14, respectively, based on tolerability and acceptable levels of transaminases. Patients were instructed to maintain a low-fat diet (<20% calories from fat) and to take dietary supplements that provided approximately 400 international units vitamin E, 210 mg alpha-linolenic acid (ALA), 200 mg linoleic acid, 110 mg eicosapentaenoic acid (EPA), and 80 mg docosahexaenoic acid (DHA) per day.

After efficacy was assessed at Week 26, patients remained on JUXTAPID for an additional 52 weeks to assess long-term safety. During this safety phase, the dose of JUXTAPID was not increased above each patient's maximum tolerated dose established during the efficacy phase, but changes to concomitant lipid-lowering treatments were allowed. Twenty-three (79%) patients completed the efficacy endpoint at Week 26, all of whom went on to complete 78 weeks of treatment.

Adverse events contributed to premature discontinuation for five patients [see Adverse Reactions (6.1) ] . The maximum tolerated doses during the efficacy period were 5 mg (10%), 10 mg (7%), 20 mg (21%), 40 mg (24%), and 60 mg (34%). The primary efficacy endpoint was percent change in LDL-C from baseline to Week 26.

At Week 26, the mean and median percent changes in LDL-C from baseline were -40% (paired t-test p<0.001) and -50%, respectively, based on the intent-to-treat population with last observation carried forward (LOCF) for patients who discontinued prematurely. The mean percent change in LDL-C from baseline through Week 26 is shown in Figure 1 for the 23 patients who completed the efficacy period. Figure 1: Mean Percent Change in LDL-C from Baseline (Week 26 Completers) Error bars represent 95% confidence intervals of the mean.

Changes in lipids and lipoproteins through the efficacy endpoint at Week 26 are presented in Table 8. Table 8: Absolute Values and Percent Changes from Baseline in Lipids and Lipoproteins PARAMETER BASELINE WEEK 26/LOCF (N=29) Mean (SD) Mean (SD) Mean % Change LDL-C, direct (mg/dL) 336 (114) 190 (104) -40 Statistically significant compared with baseline based on the pre-specified gatekeeping method for controlling Type I error among the primary and key secondary endpoints. TC (mg/dL) 430 (135) 258 (118) -36 apo B (mg/dL) 259 (80) 148 (74) -39 Non-HDL-C (mg/dL) 386 (132) 217 (113) -40 VLDL-C (mg/dL) 21 (10) 13 (9) -29 TG (mg/dL) Median values with interquartile range and median % change presented for TG.

92 [72, 128] 57 [36, 78] -45 HDL-C (mg/dL) 44 (11) 41 (13) -7 After Week 26, during the safety phase of the study, adjustments to concomitant lipid-lower… [Excerpted — this section continues on DailyMed.]

🧪 Nonclinical Toxicology ~1 min read ▾

13 NONCLINICAL TOXICOLOGY

13.1Carcinogenesis, Mutagenesis, Impairment of Fertility In a 2-year dietary carcinogenicity study in mice, lomitapide was administered at doses of 0.3, 1.5, 7.5, 15, or 45 mg/kg/day. There were statistically significant increases in the incidences of liver adenomas and carcinomas in males at doses ≥1.5 mg/kg/day (≥2-times the MRHD at 60 mg based on AUC) and in females at ≥7.5 mg/kg/day (≥10-times the human exposure at 60 mg based on AUC). Incidences of small intestinal carcinomas in males and combined adenomas and carcinomas in females were significantly increased at doses ≥15 mg/kg/day (≥23-times the human exposure at 60 mg based on AUC).

In a 2-year carcinogenicity study in rats, lomitapide was administered by oral gavage for up to 99 weeks at doses of 0.25, 1.7, or 7.5 mg/kg/day in males and 0.03, 0.35, or 2.0 mg/kg/day in females. While the design of the study was suboptimal, there were no statistically significant drug-related increases in tumor incidences at exposures up to 6-times (males) and 8-times (females) higher than human exposure at the MRHD based on AUC. Lomitapide did not exhibit genotoxic potential in a battery of studies, including the in vitro Bacterial Reverse Mutation (Ames) assay, an in vitro cytogenetics assay using primary human lymphocytes, and an oral micronucleus study in rats.

Lomitapide had no effect on fertility in rats at doses up to 5 mg/kg/day at systemic exposures estimated to be 4-times (females) and 5-times (males) higher than in humans at 60 mg based on AUC.

📄 Carcinogenesis, Mutagenesis, Impairment of Fertility ~1 min read ▾

13.1Carcinogenesis, Mutagenesis, Impairment of Fertility In a 2-year dietary carcinogenicity study in mice, lomitapide was administered at doses of 0.3, 1.5, 7.5, 15, or 45 mg/kg/day. There were statistically significant increases in the incidences of liver adenomas and carcinomas in males at doses ≥1.5 mg/kg/day (≥2-times the MRHD at 60 mg based on AUC) and in females at ≥7.5 mg/kg/day (≥10-times the human exposure at 60 mg based on AUC). Incidences of small intestinal carcinomas in males and combined adenomas and carcinomas in females were significantly increased at doses ≥15 mg/kg/day (≥23-times the human exposure at 60 mg based on AUC).

In a 2-year carcinogenicity study in rats, lomitapide was administered by oral gavage for up to 99 weeks at doses of 0.25, 1.7, or 7.5 mg/kg/day in males and 0.03, 0.35, or 2.0 mg/kg/day in females. While the design of the study was suboptimal, there were no statistically significant drug-related increases in tumor incidences at exposures up to 6-times (males) and 8-times (females) higher than human exposure at the MRHD based on AUC. Lomitapide did not exhibit genotoxic potential in a battery of studies, including the in vitro Bacterial Reverse Mutation (Ames) assay, an in vitro cytogenetics assay using primary human lymphocytes, and an oral micronucleus study in rats.

Lomitapide had no effect on fertility in rats at doses up to 5 mg/kg/day at systemic exposures estimated to be 4-times (females) and 5-times (males) higher than in humans at 60 mg based on AUC.

📄 Package Label / Principal Display Panel ~1 min read ▾

PRINCIPAL DISPLAY PANEL - 5 mg Capsule Bottle Label NDC 76431-105-01 28 capsules Juxtapid ® (lomitapide) capsules 5 mg AMRYT PHARMA PRINCIPAL DISPLAY PANEL - 5 mg Capsule Bottle Label

PRINCIPAL DISPLAY PANEL - 5 mg Capsule Bottle Carton NDC 76431-105-01 Rx Only 28 capsules Juxtapid ® (lomitapide) capsules 5 mg Attention Pharmacist: Dispense the accompanying Medication Guide to each patient. AMRYT PHARMA PRINCIPAL DISPLAY PANEL - 5 mg Capsule Bottle Carton

PRINCIPAL DISPLAY PANEL - 10 mg Capsule Bottle Label NDC 76431-110-01 28 capsules Juxtapid ® (lomitapide) capsules 10 mg AMRYT PHARMA PRINCIPAL DISPLAY PANEL - 10 mg Capsule Bottle Label

PRINCIPAL DISPLAY PANEL - 10 mg Capsule Bottle Carton NDC 76431-110-01 Rx Only 28 capsules Juxtapid ® (lomitapide) capsules 10 mg Attention Pharmacist: Dispense the accompanying Medication Guide to each patient. AMRYT PHARMA PRINCIPAL DISPLAY PANEL - 10 mg Capsule Bottle Carton

PRINCIPAL DISPLAY PANEL - 20 mg Capsule Bottle Label NDC 76431-120-01 28 capsules Juxtapid ® (lomitapide) capsules 20 mg AMRYT PHARMA PRINCIPAL DISPLAY PANEL - 20 mg Capsule Bottle Label

PRINCIPAL DISPLAY PANEL - 20 mg Capsule Bottle Carton NDC 76431-120-01 Rx Only 28 capsules Juxtapid ® (lomitapide) capsules 20 mg Attention Pharmacist: Dispense the accompanying Medication Guide to each patient. AMRYT PHARMA PRINCIPAL DISPLAY PANEL - 20 mg Capsule Bottle Carton

PRINCIPAL DISPLAY PANEL - 30 mg Capsule Bottle Label NDC 76431-130-01 28 capsules Juxtapid ® (lomitapide) capsules 30 mg AMRYT PHARMA PRINCIPAL DISPLAY PANEL - 30 mg Capsule Bottle Label

PRINCIPAL DISPLAY PANEL - 30 mg Capsule Bottle Carton NDC 76431-130-01 Rx Only 28 capsules Juxtapid ® (lomitapide) capsules 30 mg Attention Pharmacist: Dispense the accompanying Medication Guide to each patient. AMRYT PHARMA PRINCIPAL DISPLAY PANEL - 30 mg Capsule Bottle Carton

Source: FDA Structured Product Labeling, mirrored from DailyMed / openFDA. Prefer the government’s original formatting? View this label on DailyMed ↗

Medicare Part D spend CMS · PART D · 2026 (Q1)

Medicare Part D (outpatient prescription) spending for Juxtapid — the program that covers self-administered drugs. 1 manufacturer.
⚠️ Drug-level data: CMS publishes Part D spending by drug, not by NDC — these figures combine every manufacturer, strength and package size sold under the name Juxtapid. That’s a different level of aggregation than the Medicaid card above, which is specific to this exact 11-digit NDC (pack size included), so the two aren’t directly comparable.
Period
Total Part D spend
$4.69M
Claims incl. refills
61
Beneficiaries
24
Spend / beneficiary
$195,480.59
Spend / claim
$76,910.40
Trend by period
💵 About the dollar figures: spending is what Part D plans paid before confidential manufacturer rebates, so the program’s real net cost is lower. A blank patient count means fewer than 11 people — CMS hides counts that small to protect privacy. Source: CMS Medicare Quarterly Part D Spending by Drug (data.cms.gov), updated quarterly.
🛡
This drug has a REMS — JUXTAPID REMS. A Risk Evaluation & Mitigation Strategy is an FDA-required safety program. It is available only through a restricted program (certified prescribers/pharmacies, enrollment, or required monitoring). See the boxed warning & full label below, and REMS@FDA ↗.

About this NDC listing & data coverage

Finished prescription product
What data is (and isn’t) available for this NDC — tap to expand
NDC identity (package / product / labeler codes) ✓ Available
Labeler ✓ Available
Product & package description ✓ Available
Marketing category & status ✓ Available
Active ingredient / dosage form / route ✓ Available
FDA label (SPL via DailyMed) ✓ Available
Package photos ✓ Available
Inactive ingredients (structured) — Not published for this NDC The labeler did not submit a structured excipient list, or no SPL is available.
NADAC pharmacy acquisition price (CMS) — Not published for this NDC CMS publishes NADAC only for NDCs reported in its retail-pharmacy survey.
Orange Book / therapeutic-equivalence data ✓ Available
HCPCS J-code billing crosswalk — Not published for this NDC Most self-administered / retail products have no J-code — that is normal.
Medicaid utilization (CMS SDUD) — Not published for this NDC CMS reports utilization only for NDCs with Medicaid claims above its privacy threshold.
“Not published” reflects what the public FDA / CMS / NLM sources provide for this exact package code — it is a property of the data feeds, not a judgment about the product.

Questions about this listing

Why is there no price listed?
The pricing shown on our NDC pages comes from CMS NADAC, a voluntary survey of retail community pharmacy invoices. CMS does not publish a NADAC for every NDC — packages outside the retail survey (institutional and hospital products, bulk packages, discontinued items, and many OTC items) may never receive one. A missing price reflects the survey's scope, not this product's actual cost, and does not mean the product is free or unavailable.
Is the NDC printed on the package the same as the 11-digit billing NDC?
Yes, they identify this exact package in different formats. The form printed on the packaging and shown on DailyMed is the one the FDA registered. Insurance claims use a fixed 11-digit 5-4-2 format, so the short segment is padded with a leading zero and the dashes are dropped. The Identity section at the top of this page lists each form of this code.
Is this package still being marketed?
Yes, per the latest FDA NDC Directory data on this page it is currently marketed — but Amryt Pharmaceuticals DAC has reported a marketing end date of 2026-10-25, after which this package is expected to stop being marketed. The directory data on this page refreshes weekly.
Who lists this product with the FDA?
Amryt Pharmaceuticals DAC is the labeler of record for this NDC — the company under whose FDA-assigned code the package is listed. The labeler may be the manufacturer itself or a distributor marketing the product under its own code.
Do I need a prescription for this product?
This NDC is listed with FDA as a prescription product, so it is dispensed under a prescriber's order. Your pharmacist can tell you whether any over-the-counter forms of the same medication exist.
This page identifies an FDA-listed package (the NDC) and reports public regulatory and pricing data about the listing. It is reference information, not a medical recommendation — talk to your pharmacist or prescriber about your own medication.
Where does this data come from?
Listing facts (marketing category, packager status, marketing dates) from the FDA openFDA NDC Directory; label availability from DailyMed; pricing coverage from CMS NADAC; equivalence scope from the FDA Orange Book.
← 76431-0110-01 · Amryt 76431-0310-01 · Amryt →
For educational and professional reference only — not medical advice. Pricing reflects published NADAC and CMS ASP (free public data) and may differ from your acquisition cost; always verify before billing or dispensing.