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Cerdelga eliglustat 84 mg Capsule, 14-count — NDC 58468-0220-02 package photo

Cerdelga eliglustat 84 mg Capsule, 14-count

by Genzyme Corporation · 1 BLISTER PACK in 1 CARTON (58468-0220-2) / 14 CAPSULE in 1 BLISTER PACK
NDC 58468-0220-02
🏷️ FDA NDC (as labeled) 58468-0220-2 billing pads the package segment with a zero
This package
Contains14-count Pack sizes2 compare ↓
Also priced by: Part D plans $618.40/unit — full pricing hub ↓
Also comes in: 56 capsules 58468-0220-01
Rx only Brand On market Non-controlled
🗂️ Data synced 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

🆔 Identity & classification

FDA NDC (as labeled) 58468-0220-2
Product NDC 58468-0220
11-digit billing NDC 58468022002
NCPDP billing unit EA — each (per item)
RxCUI 1547225, 1547231
UNII DR40J4WA67
Application # NDA205494
SPL Set ID 819f828a-b888-4e46-83fc-94d774a28a83
Established class (EPC) Glucosylceramide Synthase Inhibitor
Mechanism of action Glucosylceramide Synthase Inhibitors; P-Glycoprotein Inhibitors; Cytochrome P450 2D6 Inhibitors
DEA schedule Non-controlled
Marketing category NDA
Marketing status On market
FDA listing status Listed (active directory)
Marketing start 2014-09-03
Route ORAL
Dosage form CAPSULE
Substance ELIGLUSTAT
GPI-14 82700040600120
GPI class Cerdelga
GCN Seq No 072709
GCN 36988
HICL code 041346
Ingredient (HICL) Eliglustat Tartrate
HIC1 code Z
Therapeutic class — broad (HIC1) Body As A Whole
HIC2 code Z1
Therapeutic class — intermediate (HIC2) Positive Effects On Wide-Spread Tissue
HIC3 code Z1G
Therapeutic class — specific (HIC3) Glucosylceramide Synthase (Gcs) Inhibitor
AHFS code 44:04.00.00
AHFS class Enzyme Inhibitors
FDB label name CERDELGA 84 MG CAPSULE
FDB brand name Cerdelga
Legend status F — Federal legend — prescription drug or device
TE code (Orange Book) AB · RLD · RS
Why two NDCs? The FDA registers this code as 58468-0220-2 — a 5-4-1 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 package segment → 58468-0220-02. 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 Glucosylceramide Synthase Inhibitor class.

Pharmacologic class Glucosylceramide Synthase Inhibitor
Drug family (ATC) Various alimentary tract and metabolism products
How it works Glucosylceramide Synthase Inhibitors, Cytochrome P450 2D6 Inhibitors, P-Glycoprotein 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.

🏭 Manufacturer & labeler

LabelerGenzyme Corporation
Application holderGENZYME CORP
FDA applicationNDA205494 (NDA)
Labeler code58468
First marketedSep 2014
Product typeHuman Prescription Drug
Portfolio33 products on file
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.

🩺 Clinical

Label name CERDELGA 84 MG CAPSULE Ingredient Eliglustat Tartrate
📗 Our plain-language guide HelloPharmacist
  • Cerdelga is used to treat Gaucher disease type 1, which is a condition where a fatty substance builds up in your liver, spleen, and bone marrow because your body lacks a key enzyme...
  • What exactly is Cerdelga used for, and how is it different from other Gaucher treatments?
  • Cerdelga is processed in your body by a liver enzyme called CYP2D6, and people naturally vary in how active that enzyme is. Your genetic type — called your metabolizer status — det...
  • Why did I need a genetic test before starting this medication?
📖 Read our full Eliglustat guide →
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.

💊 What it looks like

Color Turquoise
ShapeCapsule
ImprintGZ02
Size18 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.

💡 Tap an ingredient (hover on desktop) to see what it is and why it’s used.

  • UNII L06K8R7DQK
    A synthetic blue dye approved by the FDA for use in medications and foods. It serves as a colorant to make pills and liquids visually distinct and easier to identify.
  • UNII EX438O2MRT
    Ferric oxide yellow is a naturally occurring iron compound used as a colorant in medications. It gives tablets, capsules, and other forms a yellow or golden hue for identification and appearance.
  • UNII 2G86QN327L
    Gelatin is a protein derived from animal collagen, commonly used in medicines as a gelling agent and capsule material. It helps create soft or hard capsule shells that hold and release medication, and can also thicken liquid formulations.
  • UNII R8WTH25YS2
    Glyceryl dibehenate is a wax-like substance made from behenate fatty acids and glycerol. It functions as a binder and release-control agent in tablets and capsules, helping hold ingredients together and regulate how quickly the medicine dissolves and releases in your body.
  • UNII 3NXW29V3WO
    Hypromellose is a plant-based thickener made from cellulose. It's used in medicines as a binder to hold ingredients together, a coating for tablets, and a thickener for liquids.
  • UNII EWQ57Q8I5X
    Lactose monohydrate is a natural sugar derived from milk. It serves as a filler and binder in tablets and capsules, helping create the proper size, texture, and consistency of the medicine.
  • UNII V8A1AW0880
    Mica is a naturally occurring mineral that's ground into fine powder. It's used in medicines as a colorant and anti-caking agent to improve appearance and prevent clumping in tablets or powders.
  • UNII OP1R32D61U
    Microcrystalline cellulose is a purified form of cellulose, a natural fiber from plant sources. It acts as a binder and filler in tablets and capsules, helping hold ingredients together and give the medicine its shape and size.
  • UNII 15FIX9V2JP
    Titanium dioxide is a bright white mineral powder commonly used as a colorant and opacifying agent. It makes pills and tablets white or lighter in color and helps make coatings non-transparent.

9 inactive ingredients listed in the exact product block matched to this NDC.

Where does this data come from?
Data sourced from official FDA Structured Product Labeling (SPL) via DailyMed — ingredient classCode="IACT" elements from the exact product block matched by this NDC. Label-section narrative from DailyMed / the openFDA label index is shown separately when available.

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.

💲 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 $618.40 $8,657.57 / 14 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.

🔁 Therapeutic equivalents

ProductLabelerPackNADAC/unitTEStatusPrice vs. this
Cerdelga 84 mgthis 58468-0220-02 Genzyme 14 capsules — AB FDA listed —
About this product: this is the brand-name version. Some generic versions are approved by the FDA, but we could not confirm current pharmacy availability from our pricing/market data.
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

🏛️
2014
First FDA approval
Aug 2014
📍
2026
Currently FDA-listed
12 years listed
🛡️
2038
Latest patent/protection listed
not a guaranteed launch date
🔒Generic approved by FDA, but pharmacy availability is not confirmed

The FDA lists approved generic versions of this medicine, but that does not always mean a pharmacy can get one today. Patent rules, launch agreements, supply and pricing can affect when generics actually arrive.

🛡️ Latest patent/protection date listed: FDA patent/protection data lists protections through Dec 2038. This may affect when a full generic version becomes widely available, but it is not a guaranteed launch date.
📅 FDA approved Aug 19, 2014 AB TE-rated RLD RS ⏳ ~12.3 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 12465586 — method of use (U-3042)
US 12465586 — method of use (U-3043)
US 10888547 — method of use (U-3042)
US 10888547 — method of use (U-3043)
US 10888544 — method of use (U-3041)
US 10888544 — method of use (U-3040)
US 7196205 — drug substance
US 11458119 — drug substance
2014 2016 2018 2020 2022 2024 2026 2028 2030 2032 2034 2036 2038
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 (8)
PatentTypeUse codeExpires
US 12465586 ↗ Method of use U-3042 Jun 5, 2032
US 12465586 ↗ Method of use U-3043 Jun 5, 2032
US 10888547 ↗ Method of use U-3042 Jan 31, 2031
US 10888547 ↗ Method of use U-3043 Jan 31, 2031
US 10888544 ↗ Method of use U-3041 Dec 13, 2038
US 10888544 ↗ Method of use U-3040 Dec 13, 2038
US 7196205 ↗ Drug substance — Jun 26, 2026
US 11458119 ↗ Drug substance — Nov 24, 2030
Common questions
Is there a generic version of CERDELGA 84 MG CAPSULE?
Yes — an FDA-approved generic equivalent is listed in the FDA Orange Book for CERDELGA 84 MG CAPSULE. See the alternatives section for substitutable, lower-cost products.
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.

💊 Medicaid utilization by pack size

Medicaid (SDUD) totals over the four most recent reported quarters for every package size of this drug — handy when a specific package (e.g. a starter/titration pack) carries little or no Medicaid volume on its own.
14 capsules58468-0220-01 453 Rx · $15,314,350
14 capsules this page58468-0220-02 No Medicaid data
Drug total (last 4 qtrs): 453 Rx · 25,944 units · $15,314,350 gross reimbursed
Tap a pack size to open its page. Source: CMS State Drug Utilization Data, last 4 quarters.

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

Medicare Part D (outpatient prescription) spending for Cerdelga — 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 Cerdelga. 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
$14.47M
Claims incl. refills
450
Beneficiaries
161
Spend / beneficiary
$89,894.73
Spend / claim
$32,162.34
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.

📦 Packaging — all sizes for this product

Package NDCDescription Marketing startStatus
58468-0220-01 4 BLISTER PACK in 1 CARTON (58468-0220-1) / 14 CAPSULE in 1 BLISTER PACK 2014-09-03 Active
58468-0220-02 You're viewing this 1 BLISTER PACK in 1 CARTON (58468-0220-2) / 14 CAPSULE in 1 BLISTER PACK 2014-09-03 Active

This pack shows little to no recent Medicaid volume — a different pack size carries most fills. See all packs ↓

Pack size FAQ

What quantity is in NDC 58468-0220-02?
NDC 58468-0220-02 is a 14-count package — 1 blister pack in 1 carton / 14 capsule in 1 blister pack.
What is the difference between NDC 58468-0220-02 and NDC 58468-0220-01?
Both are Cerdelga eliglustat 84 mg Capsule — the drug itself is identical. NDC 58468-0220-02 is the 14-count package, while NDC 58468-0220-01 is the 56 capsules package.
What NDC number is used to bill for this package of Cerdelga eliglustat 84 mg Capsule?
Bill NDC 58468-0220-02 — the 11-digit billing format is 58468022002. Pharmacy and medical claims use the 11-digit form; the FDA label may print a shorter form of the same code.

🧭 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) ✓ 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 FDA registers it as 58468-0220-2, which is what is printed on the packaging and shown on DailyMed. Insurance claims use a fixed 11-digit 5-4-2 format, so the short segment is padded with a leading zero: 58468-0220-02, written without dashes as 58468022002. The Identity section at the top of this page lists every form of this code.
What do the three segments of this NDC mean?
In 58468-0220-02, the first segment (58468) is the labeler code FDA assigned to Genzyme Corporation; the middle segment (0220) identifies this specific product — its ingredient, strength, and dosage form; and the last segment (02) identifies this exact package size and type. Together they name one specific package of one specific product.
Is this package still being marketed?
Yes, per the latest FDA NDC Directory data on this page: this package is listed as actively marketed, with no marketing end date reported by Genzyme Corporation. Listing status can change — the directory data on this page refreshes weekly.
Does this product come in other package sizes?
Yes — the FDA directory lists 1 other package presentation of this same product, including 56 capsules (58468-0220-01). Each has its own NDC and its own page — see the package list near the top of this page.
Who lists this product with the FDA?
Genzyme Corporation 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.

📄 Full prescribing information FDA SPL

The complete FDA label for this product — the official prescribing information, verbatim, section by section. Jump with a chip, search within the label, or expand everything.
🎯 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
🎯 Indications and Usage 174 words ▾

1 INDICATIONS AND USAGE CERDELGA is indicated for the long-term treatment of adult patients with Gaucher disease type 1 (GD1) who are CYP2D6 extensive metabolizers (EMs), intermediate metabolizers (IMs), or poor metabolizers (PMs) as detected by an FDA-cleared test [see Dosage and Administration (2.1) ] . CERDELGA is a glucosylceramide synthase inhibitor indicated for the long-term treatment of adult patients with Gaucher disease type 1 who are CYP2D6 extensive metabolizers (EMs), intermediate metabolizers (IMs), or poor metabolizers (PMs) as detected by an FDA-cleared test.

( 1 ) Limitations of Use : CYP2D6 ultra-rapid metabolizers may not achieve adequate concentrations of CERDELGA to achieve a therapeutic effect. ( 1 ) A specific dosage cannot be recommended for CYP2D6 indeterminate metabolizers. ( 1 ) Limitations of Use : Patients who are CYP2D6 ultra-rapid metabolizers (URMs) may not achieve adequate concentrations of CERDELGA to achieve a therapeutic effect [see Clinical Studies (14) ] .

A specific dosage cannot be recommended for those patients whose CYP2D6 genotype cannot be determined (indeterminate metabolizers) [see Clinical Studies (14) ] .

⏱️ Dosage and Administration ~2 min read ▾

2 DOSAGE AND ADMINISTRATION Patient Selection ( 2.1 ) : Select patients using an FDA-cleared test for determining CYP2D6 genotype. Recommended Dosage Based on CYP2D6 Metabolizer Status ( 2.2 ) : EMs and IMs: 84 mg orally twice daily. PMs: 84 mg orally once daily.

See the Full Prescribing Information for dosing recommendations in patients receiving CYP2D6 and/or CYP3A inhibitors, or with renal or hepatic impairment. ( 2.2 , 4 , 7.1 , 8.6 , 8.7 ) Administration ( 2.4 ) : Swallow capsules whole, do not crush, dissolve or open capsules. ( 2.4 ) Avoid eating grapefruit or drinking grapefruit juice.

( 2.4 )

2.1Patient Selection Select patients with Gaucher disease type 1 based on their CYP2D6 metabolizer status. It is recommended patient genotypes be established using an FDA-cleared test for determining CYP2D6 genotype [see Indications and Usage (1) ] .

2.2Recommended Adult Dosage The recommended dosage of CERDELGA in adults is based on the patient's CYP2D6 metabolizer status. Table 1: Recommended Dosage Regimen by CYP2D6 Metabolizer Status CYP2D6 Metabolizer Status CERDELGA Dosage EMs 84 mg twice daily IMs PMs 84 mg once daily

2.3Dosage Adjustment in EMs and IMs With or Without Hepatic Impairment and Concomitant Use of CYP2D6 or CYP3A Inhibitors Reduce dosage frequency of CERDELGA 84 mg to once daily in CYP2D6 EMs and IMs with or without hepatic impairment taking CYP2D6 or CYP3A inhibitors, as shown in Table 2 [see Warnings and Precautions (5.1) , Drug Interactions (7.1) , Use in Specific Populations (8.7) ] . Table 2: Recommended Dosage of CERDELGA 84 mg Once Daily based on CYP2D6 Metabolizer, Hepatic Impairment Status, and Concomitant CYP Inhibitors CYP2D6 Metabolizer Status Hepatic Impairment Status Concomitant CYP Inhibitor EMs Without Hepatic Impairment Taking a strong or moderate CYP2D6 inhibitor Taking a strong or moderate CYP3A inhibitor Mild (Child-Pugh Class A) Hepatic Impairment Taking a weak CYP2D6 inhibitor Taking a strong, moderate, or weak CYP3A inhibitor IMs Without Hepatic Impairment Taking a strong or moderate CYP2D6 inhibitor

2.4Important Administration Instructions Swallow capsules whole, preferably with water, and do not crush, dissolve, or open the capsules. CERDELGA can be taken with or without food. Avoid the consumption of grapefruit or grapefruit juice (strong CYP3A inhibitors) with CERDELGA [see Drug Interactions (7.1) ] .

If a dose of CERDELGA is missed, take the prescribed dose at the next scheduled time; do not double the next dose. For patients currently treated with imiglucerase, velaglucerase alfa, or taliglucerase alfa, CERDELGA may be administered 24 hours after the last dose of the previous enzyme replacement therapy (ERT).

💊 Dosage Forms and Strengths 34 words ▾

3 DOSAGE FORMS AND STRENGTHS Capsules: 84 mg of eliglustat is in a capsule with a pearl blue-green opaque cap and pearl white opaque body imprinted with "GZ02" in black. Capsules: 84 mg eliglustat

⛔ Contraindications ~1 min read ▾

4 CONTRAINDICATIONS CERDELGA is contraindicated in the following patients based on CYP2D6 metabolizer status due to the risk of cardiac arrhythmias from prolongation of the PR, QTc, and/or QRS cardiac intervals. EMs Taking a strong or moderate CYP2D6 inhibitor concomitantly with a strong or moderate CYP3A inhibitor. ( 4 , 5.1 , 7.1 , 12.3 ) Moderate or severe hepatic impairment.

( 4 , 5.1 , 8.7 , 12.3 ) Mild hepatic impairment taking a strong or moderate CYP2D6 inhibitor. ( 4 , 5.1 , 8.7 , 12.3 ) IMs Taking a strong or moderate CYP2D6 inhibitor concomitantly with a strong or moderate CYP3A inhibitor. ( 4 , 5.1 , 7.1 , 12.3 ) Taking a strong CYP3A inhibitor.

( 4 , 5.1 , 7.1 , 12.3 ) Any degree of hepatic impairment. ( 4 , 5.1 , 8.7 , 12.3 ) PMs Taking a strong CYP3A inhibitor ( 4 , 5.1 , 7.1 , 12.3 ) Any degree of hepatic impairment ( 4 , 5.1 , 8.7 , 12.3 ) EMs Taking a strong or moderate CYP2D6 inhibitor concomitantly with a strong or moderate CYP3A inhibitor [see Drug Interactions (7.1) ] Moderate or severe hepatic impairment [see Use in Specific Populations (8.7) ] Mild hepatic impairment and taking a strong or moderate CYP2D6 inhibitor [see Use in Specific Populations (8.7) ] IMs Taking a strong or moderate CYP2D6 inhibitor concomitantly with a strong or moderate CYP3A inhibitor [see Drug Interactions (7.1) ] Taking a strong CYP3A inhibitor [see Drug Interactions (7.1) ] Any degree of hepatic impairment [see Use in Specific Populations (8.7) ] PMs Taking a strong CYP3A inhibitor [see Drug Interactions (7.1) ] Any degree of hepatic impairment [see Use in Specific Populations (8.7) ]

⚠️ Warnings and Cautions 179 words ▾

5 WARNINGS AND PRECAUTIONS ECG Changes and Potential for Cardiac Arrhythmias : Not recommended in patients with pre-existing cardiac disease, long QT syndrome, and concomitant use of Class IA and Class III antiarrhythmics. ( 5.1 )

5.1ECG Changes and Potential for Cardiac Arrhythmias CERDELGA is predicted to cause increases in ECG intervals (PR, QTc, and QRS) at substantially elevated eliglustat plasma concentrations and may increase the risk of cardiac arrhythmias. Use of CERDELGA is contraindicated, to be avoided, or requires dosage adjustment in patients taking CYP2D6 or CYP3A inhibitors, depending on CYP2D6 metabolizer status, type of inhibitor, or degree of hepatic impairment [see Dosage and Administration (2.3) , Contraindications (4) , Drug Interactions (7.1) ] .

Use of CERDELGA in patients with pre-existing cardiac conditions has not been studied during clinical trials. Avoid use of CERDELGA in patients with: pre-existing cardiac disease (congestive heart failure, recent acute myocardial infarction, bradycardia, heart block, ventricular arrhythmia) long QT syndrome in combination with Class IA (e.g., quinidine, procainamide) or Class III (e.g., amiodarone, sotalol) antiarrhythmic medications [see Clinical Pharmacology (12.2) ]

🤒 Adverse Reactions ~2 min read ▾

6 ADVERSE REACTIONS Most common adverse reactions (≥10%) are: fatigue, headache, nausea, diarrhea, back pain, pain in extremities, and upper abdominal pain. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Genzyme Corporation at 1-800-745-4447 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. The most common adverse reactions to CERDELGA (occurring in ≥10% of the 126 GD1 patients treated with CERDELGA across Trials 1 and 2) were fatigue, headache, nausea, diarrhea, back pain, pain in extremities, and upper abdominal pain.

The adverse reaction profile of CERDELGA is based on two controlled studies, Trials 1 and 2 [see Clinical Studies (14.1 , 14.2) ] . Table 3 presents the profile from the 9-month double-blind, randomized, placebo-controlled trial of 40 treatment-naive patients (Trial 1). Patients were between the ages of 16 and 63 on the date of the first dose of study drug, and included 20 males and 20 females.

Table 3: Adverse Reactions Occurring in ≥10% of Treatment-Naive GD1 Patients and More Frequently than Placebo (Trial 1) CERDELGA (N=20) Placebo (N=20) Adverse Reaction Patients n (%) Patients n (%) Arthralgia 9 (45) 2 (10) Headache 8 (40) 6 (30) Migraine 2 (10) 0 (0) Flatulence 2 (10) 1 (5) Nausea 2 (10) 1 (5) Oropharyngeal pain 2 (10) 1 (5) Table 4 presents the profile from the 12-month open-label, randomized, imiglucerase-controlled trial of 159 treated patients switching from enzyme replacement therapy (ERT) (Trial 2).

Patients were between the ages of 18 and 69 on the date of the first dose of CERDELGA, and included 87 females and 72 males. Table 4: Adverse Reactions Occurring in ≥5% of GD1 Patients Switching from Enzyme Replacement Therapy to CERDELGA and More Frequently than Imiglucerase (Trial 2) Trial 2 was not designed to support comparative claims for CERDELGA for the adverse reactions reported in this table. CERDELGA (N=106) Imiglucerase (N=53) Adverse Reaction Patients n (%) Patients n (%) Fatigue 15 (14) 1 (2) Headache 14 (13) 1 (2) Nausea 13 (12) 0 (0) Diarrhea 13 (12) 2 (4) Back pain 13 (12) 3 (6) Pain in extremity 12 (11) 1 (2) Upper abdominal pain 11 (10) 0 (0) Dizziness 9 (8) 0 (0) Asthenia 9 (8) 0 (0) Cough 7 (7) 2 (4) Dyspepsia 7 (7) 1 (2) Gastroesophageal reflux disease 7 (7) 0 (0) Constipation 5 (5) 0 (0) Palpitations 5 (5) 0 (0) Rash 5 (5) 0 (0) In a separate uncontrolled study, with up to 4 years of treatment in 26 naive GD1 patients, the types and incidences of adverse reactions were similar to Trials 1 and 2.

🔄 Drug Interactions ~2 min read ▾

7 DRUG INTERACTIONS See Full Prescribing Information for a list of clinically significant drug interactions. ( 7.1 , 7.2 )

7.1Effect of Other Drugs on CERDELGA Coadministration of CERDELGA with: CYP2D6 or CYP3A inhibitors may increase eliglustat concentrations which may increase the risk of cardiac arrhythmias from prolongation of the PR, QTc, and/or QRS cardiac interval [see Warnings and Precautions (5.1) , Clinical Pharmacology (12.3) ] . strong CYP3A inducers decrease eliglustat concentrations which may reduce CERDELGA efficacy [see Clinical Pharmacology (12.3) ] . See Table 5 for prevention and management of interactions with drugs affecting CERDELGA.

Use of CERDELGA is contraindicated, to be avoided, or may require dosage adjustment depending on the concomitant drug and CYP2D6 metabolizer status [see Dosage and Administration (2.2 , 2.3) , Contraindications (4) , Drug Interactions (7.1) ] . Table 5: Prevention and Management Strategies of Drug Interactions Affecting CERDELGA Based on CYP2D6 Metabolizer Status and Concomitant Interacting Drug Concomitant Drug(s) CYP2D6 Metabolizer Status EMs IMs PMs CYP2D6 Inhibitor Strong Reduce frequency of CERDELGA 84 mg to once daily Continue CERDELGA 84 mg once daily No effect of CYP2D6 inhibitor due to little or no CYP2D6 activity in CYP2D6 PMs.

Moderate Weak Continue CERDELGA 84 mg twice daily CYP3A Inhibitor Strong Reduce frequency of CERDELGA 84 mg to once daily Contraindicated Moderate Avoid coadministration Weak Continue CERDELGA 84 mg twice daily Avoid coadministration CYP2D6 Inhibitor Concomitantly with a strong CYP3A Inhibitor Strong Contraindicated Moderate CYP2D6 Inhibitor Concomitantly with a moderate CYP3A Inhibitor Strong Contraindicated Avoid coadministration Moderate CYP3A Inducer Strong Avoid coadministration

7.2Effect of CERDELGA on Other Drugs See Table 6 for clinically relevant interactions affecting P-gp or CYP2D6 substrates when coadministered with CERDELGA. Table 6: Prevention and Management Strategies of Drug Interactions Affecting Other Drugs Substrates for P-gp or CYP2D6 Clinical Impact Coadministration of CERDELGA may increase concentrations of drugs that are substrates for P-gp or CYP2D6 [see Clinical Pharmacology (12.3) ] and may increase the risk of toxicity of these drugs. Prevention or Management Digoxin Monitor serum digoxin concentrations before initiating CERDELGA.

Reduce digoxin dose by 30% and continue monitoring. Other P-gp substrates or CYP2D6 substrates Monitor therapeutic drug concentrations, as indicated, or consider reducing the dosage of the concomitant drug and titrate to clinical effect.

👥 Use in Specific Populations ~3 min read ▾

8 USE IN SPECIFIC POPULATIONS

8.1Pregnancy Risk Summary Available data on CERDELGA use in pregnant women includes 20 pregnancies that occurred during the clinical development program and a small number of post-marketing case reports. These data are not sufficient to assess drug-associated risks major birth defects, miscarriage, or adverse maternal or fetal outcomes. In animal reproduction studies in pregnant rats administered oral eliglustat during organogenesis, a spectrum of various developmental abnormalities were observed at doses 6 times the recommended human dose.

No adverse developmental outcomes were observed with oral administration of eliglustat to pregnant rabbits at dose levels 10 times the recommended human dose [see Data ] . The estimated background risk of major birth defects and miscarriage in the indicated population is unknown. All pregnancies have a background risk of birth defect, loss or other adverse outcomes.

In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2% to 4% and 15% to 20%, respectively. Clinical Considerations Disease-associated maternal and/or embryo/fetal risk Women with Gaucher disease type 1 have an increased risk of spontaneous abortion, especially if disease symptoms are not treated and controlled pre-conception and during a pregnancy. Pregnancy may exacerbate existing Gaucher disease type 1 symptoms or result in new disease manifestations.

Gaucher disease type 1 manifestations may lead to adverse pregnancy outcomes including, hepatosplenomegaly which can interfere with the normal growth of a pregnancy and thrombocytopenia which can lead to increased bleeding and possible hemorrhage. Data Animal data Reproduction studies have been performed in pregnant rats at oral doses up to 120 mg/kg/day (about 6 times the recommended human dose based on body surface area) and in pregnant rabbits at oral doses up to 100 mg/kg/day (about 10 times the recommended human dose based on body surface area) following administration of eliglustat during the period of organogenesis (gestation days 6 to 17 in the rat and 6 to 18 in the rabbit).

In rats, at 120 mg/kg/day (about 6 times the recommended human dose based on body surface area), eliglustat increased the number of late resorptions, dead fetuses and post implantation loss, reduced fetal body weight, and caused fetal cerebral variations (dilated cerebral ventricles), fetal skeletal variations (poor bone ossification) and fetal skeletal malformations (abnormal number of ribs or lumbar vertebra). Eliglustat-related effects on fetal rats were observed in association with signs of maternal toxicity. Eliglustat did not cause fetal harm in rabbits at oral doses up to 100 mg/kg/day (about 10 times the recommended human dose based on body surface area).

Mild maternal toxicity was observed at the 100 mg/kg/day dose. In a pre and postnatal development study in rats (dosed daily from gestation day 6 to postpartum day 21), eliglustat did not show any significant adverse effects on pre and postnatal development at doses up to 100 mg/kg/day (about 5 times the recommended human dose based on body surface area).

8.2Lactation Risk Summary There are no human data available on the presence of eliglustat in human milk, the effects on the breastfed infant, or the effects on milk production. Eliglustat and its metabolites were present in the milk of lactating rats [see Data ] . When a drug is present in animal milk, it is likely that the drug will be present in human milk.

The developmental and health benefits of breastfeeding should be considered along with the mother's clinical need for CERDELGA and any potential adverse effects on the breastfed child from CERDELGA or from the underlying maternal condition. Data In a milk excretion study in the rat, a single oral dose of 30 mg/kg [ 14 C]-labeled eliglustat was administered to lactating female rats at day 11 postpartum. Approximately…

🤰 Pregnancy ~2 min read ▾

8.1Pregnancy Risk Summary Available data on CERDELGA use in pregnant women includes 20 pregnancies that occurred during the clinical development program and a small number of post-marketing case reports. These data are not sufficient to assess drug-associated risks major birth defects, miscarriage, or adverse maternal or fetal outcomes. In animal reproduction studies in pregnant rats administered oral eliglustat during organogenesis, a spectrum of various developmental abnormalities were observed at doses 6 times the recommended human dose.

No adverse developmental outcomes were observed with oral administration of eliglustat to pregnant rabbits at dose levels 10 times the recommended human dose [see Data ] . The estimated background risk of major birth defects and miscarriage in the indicated population is unknown. All pregnancies have a background risk of birth defect, loss or other adverse outcomes.

In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2% to 4% and 15% to 20%, respectively. Clinical Considerations Disease-associated maternal and/or embryo/fetal risk Women with Gaucher disease type 1 have an increased risk of spontaneous abortion, especially if disease symptoms are not treated and controlled pre-conception and during a pregnancy. Pregnancy may exacerbate existing Gaucher disease type 1 symptoms or result in new disease manifestations.

Gaucher disease type 1 manifestations may lead to adverse pregnancy outcomes including, hepatosplenomegaly which can interfere with the normal growth of a pregnancy and thrombocytopenia which can lead to increased bleeding and possible hemorrhage. Data Animal data Reproduction studies have been performed in pregnant rats at oral doses up to 120 mg/kg/day (about 6 times the recommended human dose based on body surface area) and in pregnant rabbits at oral doses up to 100 mg/kg/day (about 10 times the recommended human dose based on body surface area) following administration of eliglustat during the period of organogenesis (gestation days 6 to 17 in the rat and 6 to 18 in the rabbit).

In rats, at 120 mg/kg/day (about 6 times the recommended human dose based on body surface area), eliglustat increased the number of late resorptions, dead fetuses and post implantation loss, reduced fetal body weight, and caused fetal cerebral variations (dilated cerebral ventricles), fetal skeletal variations (poor bone ossification) and fetal skeletal malformations (abnormal number of ribs or lumbar vertebra). Eliglustat-related effects on fetal rats were observed in association with signs of maternal toxicity. Eliglustat did not cause fetal harm in rabbits at oral doses up to 100 mg/kg/day (about 10 times the recommended human dose based on body surface area).

Mild maternal toxicity was observed at the 100 mg/kg/day dose. In a pre and postnatal development study in rats (dosed daily from gestation day 6 to postpartum day 21), eliglustat did not show any significant adverse effects on pre and postnatal development at doses up to 100 mg/kg/day (about 5 times the recommended human dose based on body surface area).

🧒 Pediatric Use 13 words ▾

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

🧓 Geriatric Use 41 words ▾

8.5Geriatric Use Clinical studies of CERDELGA did not include sufficient numbers of subjects aged 65 and over to determine whether they respond differently from younger subjects. Clinical experience has not identified differences in responses between the elderly and younger patients.

🆘 Overdosage 102 words ▾

10 OVERDOSAGE The highest eliglustat plasma concentration experienced to date occurred in a single-dose, dose escalation study in healthy subjects, in a subject taking a dose equivalent to approximately 21 times the recommended dose for GD1 patients. At the time of the highest plasma concentration (59-fold higher than normal therapeutic conditions), the subject experienced dizziness marked by disequilibrium, hypotension, bradycardia, nausea, and vomiting. In the event of acute overdose, the patient should be carefully observed and given symptomatic and supportive treatment.

Hemodialysis is unlikely to be beneficial given that eliglustat has a large volume of distribution [see Clinical Pharmacology (12.3) ] .

🧬 Clinical Pharmacology ~3 min read ▾

12 CLINICAL PHARMACOLOGY

12.1Mechanism of Action Gaucher disease is caused by a deficiency of the lysosomal enzyme acid β-glucosidase. Acid β-glucosidase catalyzes the conversion of the sphingolipid glucocerebroside into glucose and ceramide. The enzymatic deficiency causes an accumulation of glucosylceramide (GL-1) primarily in the lysosomal compartment of macrophages, giving rise to foam cells or "Gaucher cells." The clinical features of this lysosomal storage disorder (LSD) are reflective of the accumulation of Gaucher cells in the reticuloendothelial system (liver, spleen, bone marrow, and other organs).

The accumulation of Gaucher cells in the liver, spleen, and bone marrow leads to organomegaly and skeletal disease. Presence of Gaucher cells in the bone marrow and spleen leads to clinically significant anemia and thrombocytopenia. CERDELGA is a specific inhibitor of glucosylceramide synthase (IC 50 =10 ng/mL) and acts as a substrate reduction therapy for GD1 by reducing the production of GL-1.

By reducing GL-1 production, CERDELGA alleviates the accumulation of GL-1 in the target organs.

12.2Pharmacodynamics Effects on spleen and liver volume, hemoglobin, and platelets increased with increasing steady-state average trough concentrations of eliglustat ranging up to 14 ng/mL in treatment naive patients in Trial 1. In patients previously treated with enzyme-replacement therapy in Trial 2 [see Clinical Studies (14.2) ] , no clinically relevant exposure-response relationship was observed. Cardiac Electrophysiology Concentration-related increases were observed for the placebo-corrected change from baseline in the PR, QRS, and QTc intervals.

At the mean peak concentration of 237 ng/mL at a dose of 800 mg eliglustat tartrate (8 times the recommended dose), CERDELGA did not prolong the QT/QTc interval to any clinically relevant extent. However, pharmacokinetic/pharmacodynamic modeling predicts mean (upper bound of the 95% one-sided confidence interval) increases in the PR, QRS, and QTcF intervals of 22 (26), 7 (10), and 13 (19) msec, respectively, at eliglustat plasma concentration of 500 ng/mL [see Warnings and Precautions (5.1) ] .

12.3Pharmacokinetics Absorption The oral bioavailability of eliglustat was less than 5% in CYP2D6 EMs following a single 84 mg dose of CERDELGA. In CYP2D6 EMs, the eliglustat pharmacokinetics is time-dependent and the systemic exposure increases in a more than dose-proportional manner over the dose range of 42 to 294 mg (0.5 to 3.5 times the recommended dosage). In addition, after multiple oral doses of 84 mg twice daily in EMs, eliglustat systemic exposure (AUC 0–12 ) increased up to about 2-fold at steady state compared to after the first dose (AUC 0–∞ ).

The pharmacokinetics of eliglustat in CYP2D6 PMs is expected to be linear and time-independent. Compared to EMs, the systemic exposure following 84 mg twice daily at steady state is 7-fold to 9-fold higher in PMs. Dosing of CERDELGA 84 mg once daily has not been studied in PMs.

The predicted C max and AUC 0–24hr in PMs using a physiologically based pharmacokinetic (PBPK) model with 84 mg once daily were 75 ng/mL and 956 hr∙ng/mL, respectively. Table 7 describes the pharmacokinetic parameters for eliglustat in healthy subjects following multiple doses of 84 mg CERDELGA twice daily. Table 7: Pharmacokinetic Parameters for Eliglustat following Multiple Doses of 84 mg CERDELGA Twice Daily Parameter CYP2D6 Metabolizer Status EMs (n=96) IMs (n=1) PMs 84 mg twice daily is not the recommended dosage in PMs [see Dosage and Administration (2.2) ] .

(n=9) C max (ng/mL) Range of the mean (CV%) values from multiple studies. 12.1 (42%) to 25.0 (141%) 44.6 113 (32%) to 137 (40%) AUC tau (ng∙hr/mL) 76.3 (37%) to 143 (160%) 306 922 (33%) to 1057 (38%) Median T max (hr) [min to max] Range of the median time to reach maximum plasma concentration (T max ) from multiple studies. 1.5 [0.5 to 3.0] to 2 [1.5 to 2.1] 2 3 [2 to 4] Administration of CERDELGA with a high…

🧬 Mechanism of Action 158 words ▾

12.1Mechanism of Action Gaucher disease is caused by a deficiency of the lysosomal enzyme acid β-glucosidase. Acid β-glucosidase catalyzes the conversion of the sphingolipid glucocerebroside into glucose and ceramide. The enzymatic deficiency causes an accumulation of glucosylceramide (GL-1) primarily in the lysosomal compartment of macrophages, giving rise to foam cells or "Gaucher cells." The clinical features of this lysosomal storage disorder (LSD) are reflective of the accumulation of Gaucher cells in the reticuloendothelial system (liver, spleen, bone marrow, and other organs).

The accumulation of Gaucher cells in the liver, spleen, and bone marrow leads to organomegaly and skeletal disease. Presence of Gaucher cells in the bone marrow and spleen leads to clinically significant anemia and thrombocytopenia. CERDELGA is a specific inhibitor of glucosylceramide synthase (IC 50 =10 ng/mL) and acts as a substrate reduction therapy for GD1 by reducing the production of GL-1.

By reducing GL-1 production, CERDELGA alleviates the accumulation of GL-1 in the target organs.

📦 How Supplied / Storage and Handling 111 words ▾

16 HOW SUPPLIED/STORAGE AND HANDLING CERDELGA is supplied as 84 mg eliglustat in a capsule with a pearl blue-green opaque cap and pearl white opaque body imprinted with "GZ02" in black. CERDELGA 84 mg capsules are supplied as: NDC 58468-0220-1 – Carton containing 4 packs of capsules (56 capsules total). Each pack is composed of 1 blister card of 14 capsules and a cardboard wallet.

NDC 58468-0220-2 – Carton containing 1 pack of capsules (14 capsules total). Each pack is comprised of 1 blister card of 14 capsules and a cardboard wallet. Store at 68°F–77°F (20°C–25°C) with excursions permitted between 59°F and 86°F (15°C to 30°C) [see USP Controlled Room Temperature].

📦 Storage and Handling 19 words ▾

Store at 68°F–77°F (20°C–25°C) with excursions permitted between 59°F and 86°F (15°C to 30°C) [see USP Controlled Room Temperature].

📋 Description 112 words ▾

11 DESCRIPTION CERDELGA (eliglustat) capsules contain eliglustat tartrate, which is a small molecule inhibitor of glucosylceramide synthase that resembles the ceramide substrate for the enzyme, with the chemical name N-((1 R ,2 R )-1-(2,3-dihydrobenzo[b][1,4]dioxin-6-yl)-1-hydroxy-3-(pyrrolidin-1-yl)propan-2-yl)octanamide (2 R ,3 R )-2,3-dihydroxysuccinate. Its molecular weight is 479.59, and the empirical formula is C 23 H 36 N 2 O 4 +½(C 4 H 6 O 6 ) with the following chemical structure: Each capsule of CERDELGA for oral use contains 84 mg of eliglustat (equivalent to 100 mg of eliglustat tartrate).

The inactive ingredients are candurin silver fine, FD&C blue 2, gelatin, glyceryl behenate, hypromellose, lactose monohydrate, microcrystalline cellulose, and yellow iron oxide. Chemical Structure

💬 Information for Patients 201 words ▾

17 PATIENT COUNSELING INFORMATION Advise the patient to read the FDA-approved patient labeling (Medication Guide). Drug Interactions Advise patients to discuss all the medications they are taking, including any herbal supplements or vitamins with their healthcare provider [see Contraindications (4) , Warnings and Precautions (5) , Drug Interactions (7) ] . ECG Changes and Potential for Cardiac Arrhythmias Advise patients to inform their healthcare provider of the following: history of congestive heart failure; recent acute myocardial infarction; bradycardia; heart block; ventricular arrhythmia; and long QT syndrome [see Warnings and Precautions (5.1) ] .

Advise patients to inform their healthcare provider if they develop new symptoms such as palpitations, fainting, and dizziness. Administration Instructions [See Dosage and Administration (2.4) .] Advise patients: Swallow capsules whole, preferably with water, and do not crush, dissolve, or open the capsules. CERDELGA can be taken with or without food.

If a dose of CERDELGA is missed, take the prescribed dose at the next scheduled time; do not double the next dose. Avoid consumption of grapefruit or its juice. For patients currently treated with imiglucerase, velaglucerase alfa, or taliglucerase alfa, CERDELGA may be administered 24 hours after the last dose of the previous enzyme replacement therapy (ERT).

💬 Medication Guide ~3 min read ▾

MEDICATION GUIDE CERDELGA ® (sir-DEL-guh) (eliglustat) capsules This Medication Guide has been approved by the U.S. Food and Drug Administration. Issued: January 2024 What is the most important information I should know about CERDELGA?

CERDELGA can affect the way other medicines work and other medicines can affect how CERDELGA works. Using CERDELGA with other medicines or herbal supplements may cause an increased risk of side effects. Especially tell your doctor if you take: St.

John's Wort (Hypericum perforatum) Medicine for: Fungal infections Tuberculosis Seizures Heart conditions or high blood pressure Depression or other mental health problems If you take any medicines for the conditions listed above, your doctor may need to prescribe a different medicine, change your dose of other medicines, or change your dose of CERDELGA. Tell your doctor about any new medicines before you start taking them. What is CERDELGA?

CERDELGA is a prescription medicine used for the long-term treatment of Gaucher disease type 1 (GD1) in adults. CERDELGA is not used in certain people with Gaucher disease type 1. Your doctor will perform a test to make sure that CERDELGA is right for you.

It is not known if CERDELGA is safe and effective in children. What should I tell my doctor before taking CERDELGA? Before taking CERDELGA, tell your doctor about all of your medical conditions, including if you: have heart problems, including a condition called long QT syndrome have a history of a heart attack have kidney or liver problems are pregnant or planning to become pregnant.

It is not known if CERDELGA will harm your unborn baby. are breastfeeding or planning to breastfeed. It is not known if CERDELGA passes into your breast milk. You and your doctor will decide if you should take CERDELGA or breastfeed.

You should not do both. Tell your doctor about all of the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. See " What is the most important information I should know about CERDELGA? " How should I take CERDELGA?

Take CERDELGA exactly as your doctor tells you to take it. Your doctor may change your dose if needed. Take CERDELGA capsules whole, preferably with water.

Do not open, crush, or dissolve capsules before swallowing. CERDELGA can be taken with or without food. If you miss a dose of CERDELGA, take the next dose at the usual time.

Do not take two doses of CERDELGA at the same time. If you take too much CERDELGA, call your doctor or go to the nearest hospital emergency room right away. What should I avoid while taking CERDELGA?

Avoid eating or drinking grapefruit products while taking CERDELGA. Grapefruit products can increase the amount of CERDELGA in your body. What are the possible side effects of CERDELGA?

See " What is the most important information I should know about CERDELGA? " CERDELGA, used with certain other medicines, may cause changes in the electrical activity of your heart (ECG changes) and irregular heart beat (arrhythmias). Tell your doctor if you have new symptoms such as palpitations, fainting, or dizziness. The most common side effects of CERDELGA include: tiredness, headache, nausea, diarrhea, and pain in the arms, legs, back, or stomach (abdomen).

Tell your doctor if you have any side effect that bothers you or that does not go away. These are not all the possible side effects of CERDELGA. Call your doctor for medical advice about side effects.

You may report side effects to FDA at 1-800-FDA-1088. How should I store CERDELGA? Store CERDELGA at room temperature between 68°F-77 °F (20°C-25 °C).

Keep CERDELGA and all medicines out of reach of children. General information about the safe and effective use of CERDELGA. Medicines are sometimes prescribed for purposes other than those listed in a Medication Guide.

Do not use CERDELGA for a condition for which it was not prescribed. Do not give CERDELGA to other people, even if they have the same symptoms you have. It may harm…

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