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OPFOLDA miglustat 65 mg Capsule — NDC 71904-0300-01 package photo

OPFOLDA miglustat 65 mg Capsule

by AMICUS THERAPEUTICS US, LLC · 4 BOTTLE, PLASTIC in 1 BOTTLE, PLASTIC (71904-300-01) / 1 CAPSULE in 1 BOTTLE, PLASTIC
NDC 71904-0300-01
🏷️ FDA NDC (as labeled) 71904-300-01 billing pads the product segment with a zero
This package
Contains1 capsule in 1 bottle, plastic Pack sizes3 compare ↓
Also comes in: 1 capsule 71904-0300-02 1 capsule 71904-0300-03
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) 71904-300-01
Product NDC 71904-300
11-digit billing NDC 71904030001
NCPDP billing unit EA — each (per item)
RxCUI 2667426, 2667432
UNII ADN3S497AZ
UPC 0371904300016, 0371904300023, 0371904300030
Application # NDA215211
SPL Set ID d7c38298-37db-5a80-e053-2995a90aee40
Established class (EPC) Glucosylceramide Synthase Inhibitor; Enzyme Stabilizer
Mechanism of action Glucosylceramide Synthase Inhibitors; Enzyme Stabilizers
DEA schedule Non-controlled
Marketing category NDA
Marketing status On market
FDA listing status Listed (active directory)
Marketing start 2023-10-11
Route ORAL
Dosage form CAPSULE
Substance MIGLUSTAT
GPI-14 30907760000120
GCN Seq No 085343
GCN 54801
HICL code 025098
Ingredient (HICL) Miglustat
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 OPFOLDA 65 MG CAPSULE
FDB brand name Opfolda
Legend status F — Federal legend — prescription drug or device
Why two NDCs? The FDA registers this code as 71904-300-01 — 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 → 71904-0300-01. 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 Glucosylceramide Synthase Inhibitor class.

Pharmacologic class Glucosylceramide Synthase Inhibitor, Enzyme Stabilizer
Drug family (ATC) Various alimentary tract and metabolism products
How it works Glucosylceramide Synthase Inhibitors, Enzyme Stabilizers
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

LabelerAMICUS THERAPEUTICS US, LLC
Application holderAMICUS THERAPEUTICS US LLC
FDA applicationNDA215211 (NDA)
Labeler code71904
First marketedOct 2023
Product typeHuman Prescription Drug
Portfolio3 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 OPFOLDA 65 MG CAPSULE Ingredient Miglustat
📖 What it is MedlinePlus · NLM

Miglustat is used to treat Gaucher disease type 1 (a condition in which a certain fatty substance is not broken down normally in the body and instead builds up in some organs and causes liver, spleen, bone, and blood problems). Miglustat is also used in combination with another medication known as cipaglucosidase alfa-atga (Pombilti) to treat late onset Pompe disease (a genetic condition that causes deficiency in lysosomal acid alpha-glucosidase, which can lead to muscle weakness and wasting) in adult patients who weigh 92 pounds (40 kg) or more and who have not been improving on their enzyme...

Read the full MedlinePlus article ↗
📗 Our plain-language guide HelloPharmacist
  • Miglustat (also sold as Yargesa or Zavesca) treats adults with mild to moderate type 1 Gaucher disease — a rare condition where a missing enzyme lets a fatty substance build up in...
  • For type 1 Gaucher disease, miglustat capsules are generally taken three times a day. You can take them with or without food — food slows absorption slightly but doesn't change how...
  • How should I take my miglustat capsules?
  • Yes — diarrhea is very common with miglustat, affecting up to 89% of patients in studies. It happens because miglustat interferes with the digestion of certain sugars in your gut....
📖 Read our full Miglustat 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.

🧪 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.

💲 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 · quarterly No Part D plan price is available for this NDC in our data.
ℹ️
No price is published for this exact package yet. CMS surveys NADAC per package size, so a different pack of the same drug often has one.
Try another pack size: 1 capsule 1 capsule
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
Opfolda 65 mgthis 71904-0300-01 AMICUS 1 capsule — — 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

🏛️
2023
First FDA approval
Sep 2023
📍
2026
Currently FDA-listed
3 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 Sep 2038. This may affect when a full generic version becomes widely available, but it is not a guaranteed launch date.
📅 FDA approved Sep 28, 2023 RLD RS ⏳ ~12 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 12419937 — method of use (U-3726)
US 12414985 — method of use (U-3726)
US 12246062 — method of use (U-3726)
US 11753632 — method of use (U-3726)
US 11278601 — method of use (U-3726)
US 11278599 — method of use (U-3726)
US 10961522 — method of use (U-3726)
US 10857212 — method of use (U-3726)
US 10512677 — method of use (U-3726)
US 10208299 — drug substance (U-3726)
Exclusivity NP
2023 2025 2027 2029 2031 2033 2035 2037
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 (10)
PatentTypeUse codeExpires
US 12419937 ↗ Method of use U-3726 Mar 7, 2033
US 12414985 ↗ Method of use U-3726 Dec 29, 2036
US 12246062 ↗ Method of use U-3726 Sep 16, 2038
US 11753632 ↗ Method of use U-3726 Sep 30, 2035
US 11278601 ↗ Method of use U-3726 Dec 29, 2036
US 11278599 ↗ Method of use U-3726 Mar 7, 2033
US 10961522 ↗ Method of use U-3726 Sep 30, 2035
US 10857212 ↗ Method of use U-3726 Aug 12, 2037
US 10512677 ↗ Method of use U-3726 Mar 7, 2033
US 10208299 ↗ Drug substance U-3726 Sep 30, 2035
FDA exclusivity
CodeWhat it grantsExpires
NPNew ProductSep 28, 2026
Common questions
Is there a generic version of OPFOLDA 65 MG CAPSULE?
Yes — an FDA-approved generic equivalent is listed in the FDA Orange Book for OPFOLDA 65 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.

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

Medicare Part D (outpatient prescription) spending for Opfolda — 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 Opfolda. 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
$18.7K
Claims incl. refills
75
Beneficiaries
23
Spend / beneficiary
$813.11
Spend / claim
$249.35
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.

🔬 Reported adverse events (FAERS)

Read carefully: FAERS reports are voluntary and unverified. Counts are not incidence, do not establish causation, are subject to reporting bias, and cannot be used to compare one drug to another. Shown for signal context only. Reports for Miglustat — the ingredient across all brands.

Top reported reactions

Diarrhoea164
Death158
Seizure110
Weight Decreased98
Niemann-pick Disease97
Pneumonia91
Disease Progression84

Reporter sex

1,154 reports

Serious outcomes

Death319
Reports over time (by year) — tap or hover for the count & year
2019 2021 2023 2026 115 28
Most recent year is provisional (FAERS lags ~3 months).
Where does this data come from?
Adverse-event reports from the FDA Adverse Event Reporting System (FAERS) via openFDA. FAERS reports are voluntary and unverified — counts are not incidence and don’t establish causation.

📦 Packaging — all sizes for this product

Package NDCDescription Marketing startStatus
71904-0300-01 You're viewing this 4 BOTTLE, PLASTIC in 1 BOTTLE, PLASTIC (71904-300-01) / 1 CAPSULE in 1 BOTTLE, PLASTIC 2023-10-11 Active
71904-0300-02 24 BOTTLE, PLASTIC in 1 BOTTLE, PLASTIC (71904-300-02) / 1 CAPSULE in 1 BOTTLE, PLASTIC 2023-10-11 Active
71904-0300-03 100 BOTTLE, PLASTIC in 1 BOTTLE, PLASTIC (71904-300-03) / 1 CAPSULE in 1 BOTTLE, PLASTIC 2023-10-11 Active

Pack size FAQ

What quantity is in NDC 71904-0300-01?
NDC 71904-0300-01 is listed by the FDA — 4 bottle, plastic in 1 bottle, plastic / 1 capsule in 1 bottle, plastic.
What NDC number is used to bill for this package of OPFOLDA miglustat 65 mg Capsule?
Bill NDC 71904-0300-01 — the 11-digit billing format is 71904030001. 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) — 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 FDA registers it as 71904-300-01, 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: 71904-0300-01, written without dashes as 71904030001. 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 71904-0300-01, the first segment (71904) is the labeler code FDA assigned to AMICUS THERAPEUTICS US, LLC; the middle segment (0300) identifies this specific product — its ingredient, strength, and dosage form; and the last segment (01) 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 AMICUS THERAPEUTICS US, LLC. 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 2 other package presentations of this same product, including 1 capsule (71904-0300-02), 1 capsule (71904-0300-03). 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?
AMICUS THERAPEUTICS US, LLC 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 👥 Use in Specific Populations 🤰 Pregnancy 🧒 Pediatric Use 🧓 Geriatric Use 🧬 Clinical Pharmacology 🧬 Mechanism of Action 📦 How Supplied / Storage and Handling 📋 Description 💬 Information for Patients
🎯 Indications and Usage 89 words ▾

1 INDICATIONS AND USAGE OPFOLDA is indicated, in combination with Pombiliti, for the treatment of adult patients with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) weighing ≥40 kg and who are not improving on their current enzyme replacement therapy (ERT). OPFOLDA is an enzyme stabilizer indicated, in combination with Pombiliti, a hydrolytic lysosomal glycogen-specific enzyme, for the treatment of adult patients with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) weighing ≥40 kg and who are not improving on their current enzyme replacement therapy (ERT).

( 1 )

⏱️ Dosage and Administration ~2 min read ▾

2 DOSAGE AND ADMINISTRATION Verify pregnancy status in females of reproductive potential prior to initiating treatment. (2.1) Administer OPFOLDA in combination with Pombiliti. (2.2) Recommended OPFOLDA dosage (based on actual body weight), administered orally every other week, is: (2.2) 260 mg for patients weighing ≥50 kg.

195 mg for patients weighing ≥40 kg to <50 kg. Start OPFOLDA in combination with Pombiliti 2 weeks after the last ERT dose. (2.2) Take OPFOLDA with an unsweetened beverage approximately 1 hour before the start of Pombiliti infusion; do not consume other beverages or food for at least 2 hours prior to and 2 hours after taking OPFOLDA.

(2.2) Missed dose: If the OPFOLDA dosage is missed, Pombiliti should not be administered and treatment should be rescheduled at least 24 hours after OPFOLDA was last taken. If OPFOLDA in combination with Pombiliti are both missed, re-start treatment as soon as possible. (2.2) See full prescribing information for recommended OPFOLDA dosage in patients with renal impairment.

(2.3)

2.1Pregnancy Evaluation Prior to Initiating Treatment Verify the pregnancy status of females of reproductive potential prior to initiating OPFOLDA in combination with Pombiliti [see Use in Specific Populations (8.1, 8.3) ] .

2.2Recommended Dosage and Administration OPFOLDA must be administered in combination with Pombiliti (see Figure 1 for the dosing timeline). Refer to the Pombiliti Prescribing Information for Pombiliti dosage and administration recommendations. The recommended dosage of OPFOLDA is based on actual body weight.

For patients weighing: ≥50 kg, the recommended dosage is 260 mg orally every other week. ≥40 kg to <50 kg, the recommended dosage is 195 mg orally every other week. Start OPFOLDA in combination with Pombiliti 2 weeks after the last ERT dose. Take OPFOLDA approximately 1 hour before intravenous administration of Pombiliti.

Swallow the OPFOLDA capsules whole only with unsweetened beverages (e.g., water, tea or coffee with no cream, sugar, or sweeteners). Do not consume other beverages or food for at least 2 hours prior to and 2 hours after administration of OPFOLDA. Missed Dose If the OPFOLDA dosage is missed, Pombiliti should not be administered and treatment should be rescheduled at least 24 hours after OPFOLDA was last taken.

If OPFOLDA in combination with Pombiliti are both missed, re-start treatment as soon as possible. Figure 1. Dosing Timeline Figure 1

2.3Recommended Dosage in Patients with Renal Impairment The recommended dosage of OPFOLDA in patients with moderate or severe renal impairment is shown in Table 1 [see Clinical Pharmacology (12.3) ] . Table 1. Recommended OPFOLDA Dosage in Patients with Moderate or Severe Renal Impairment ∗ Renal function classified by CLcr (creatinine clearance) based on the Cockcroft-Gault equation.

Patient Weight Moderate Renal Impairment∗(CLcr 30-59 mL/minute) Severe Renal Impairment∗ (CLcr 15-29 mL/minute) ≥50 kg 195 mg 195 mg ≥40 kg to <50 kg 130 mg 130 mg For patients with mild renal impairment (creatinine clearance based on the Cockcroft-Gault equation, CLcr 60-89 mL/minute), the recommended OPFOLDA dosage is the same as for patients with normal renal function.

💊 Dosage Forms and Strengths 31 words ▾

3 DOSAGE FORMS AND STRENGTHS Capsules: 65 mg of miglustat, white opaque hard gelatin capsule with grey opaque cap with “AT2221” printed in black on the body. Capsules: 65 mg (3)

⛔ Contraindications 24 words ▾

4 CONTRAINDICATIONS OPFOLDA in combination with Pombiliti is contraindicated in Pregnancy [see Use in Specific Populations (8.1 )] Pregnancy. ( 4, 5.1, 8.1 )

⚠️ Warnings and Cautions ~1 min read ▾

5 WARNINGS AND PRECAUTIONS Embryo-Fetal Toxicity: May cause embryo-fetal harm. Advise females of reproductive potential of the potential risk to a fetus and to use effective contraception during treatment and for at least 60 days after the last dose. ( 4, 5.1, 8.1, 8.3 ) Risks Associated with Pombiliti: Refer to the Pombiliti Prescribing Information for a description of additional risks for Pombiliti. (5.2)

5.1Embryo-Fetal Toxicity Based on findings from animal reproduction studies, OPFOLDA in combination with Pombiliti may cause embryo-fetal harm when administered to a pregnant female and is contraindicated during pregnancy. In a rabbit embryo-fetal development study, great vessel and cardiac malformations were increased in offspring of pregnant rabbits treated with oral miglustat in combination with cipaglucosidase alfa-atga at 3-fold and 16-fold, respectively, the maximum recommended human dose (MRHD) based on plasma AUC exposure.

Verify the pregnancy status in females of reproductive potential prior to initiating treatment with OPFOLDA in combination with Pombiliti. Advise females of reproductive potential to use effective contraception during treatment with OPFOLDA in combination with Pombiliti and for at least 60 days after the last dose [see Use in Specific Populations (8.1, 8.3) ] .

5.2Risks Associated with Pombiliti OPFOLDA must be administered in combination with Pombiliti. Refer to the Pombiliti Prescribing Information for a description of additional risks for Pombiliti including, but not limited to, the warnings and precautions for Pombiliti.

🤒 Adverse Reactions ~3 min read ▾

6 ADVERSE REACTIONS Most common adverse reactions ≥5% are headache, diarrhea, fatigue, nausea, abdominal pain, and pyrexia. (6.1) To report SUSPECTED ADVERSE REACTIONS, contact Amicus Therapeutics at 1-877-4AMICUS 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. Adverse Reactions from the Pooled Clinical Trials Including Trial 1 The pooled safety analysis from 3 clinical trials included 151 adult patients with late-onset Pompe disease (LOPD) treated with OPFOLDA in combination with Pombiliti including: 85 patients in the randomized, double-blind, active-controlled trial in adults (Trial 1) [see Clinical Studies (14) ] , 37 patients in the open-label extension trial where patients switched from a non‑U.S.‑approved alglucosidase alfa product [see Clinical Studies (14) ] to OPFOLDA in combination with Pombiliti, 29 patients in an open-label trial.

The total median duration of exposure in these trials was 21 months, with 120 patients having at least 12 months exposure to OPFOLDA in combination with Pombiliti. In these trials, 78% (n=117) of the patients received previous ERT (ERT‑experienced) with a mean treatment duration of 7.7 years. The most common adverse reactions (≥5%) reported in the pooled safety population of patients treated with OPFOLDA in combination with Pombiliti in the 3 clinical trials were headache, diarrhea, fatigue, nausea, abdominal pain, and pyrexia.

Adverse Reactions from Trial 1 Trial 1 (a randomized, double-blind, active-controlled trial) included 123 adult patients with LOPD who were randomized in a 2:1 ratio to receive treatment with OPFOLDA in combination with Pombiliti or a non-U.S.-approved alglucosidase alfa product with placebo [see Clinical Studies (14) ]. The duration of exposure was similar for both treatment groups (overall mean exposure of 12 months). Most patients (77%) were ERT‑experienced, and a majority of patients in both treatment groups had >5 years of prior treatment with ERT (69% and 63% of patients in the OPFOLDA in combination with Pombiliti group and the non-U.S.-approved alglucosidase alfa product with placebo group, respectively).

The most common adverse reactions (≥5%) reported in the patients who received OPFOLDA in combination with Pombiliti in Trial 1 were headache and diarrhea. Table 2 summarizes frequent adverse reactions that occurred in patients treated with OPFOLDA in combination with Pombiliti in Trial 1. Trial 1 was not designed to demonstrate a statistically significant difference in the incidence of adverse reactions in the OPFOLDA in combination with Pombiliti and the non-U.S.-approved alglucosidase alfa product with placebo groups.

Table 2. Adverse Reactions that Occurred in Adults with LOPD at an Incidence of ≥2% in Trial 1 LOPD: late-onset Pompe disease ∗ Headache included migraine and migraine with aura. † Rash included erythematous rash and macular rash. ‡ Abdominal pain included upper and lower abdominal pain. § Tachycardia included sinus tachycardia. ¶ Urticaria included mechanical urticaria and urticarial rash. Adverse Reaction OPFOLDA in Combination with Pombiliti (n=85) N (%) A Non-U.S.-Approved Alglucosidase alfa Product with Placebo (n=38) N (%) Headache ∗ 7 (8.2) 3 (7.9) Diarrhea 5 (5.9) 2 (5.3) Dizziness 4 (4.7) 2 (5.3) Dyspnea 3 (3.5) 0 Abdominal distention 3 (3.5) 2 (5.3) Pyrexia 3 (3.5) 1 (2.6) Rash † 3 (3.5) 0 Abdominal pain ‡ 2 (2.4) 4 (10.5) Nausea 2 (2.4) 5 (13.2) Chills 2 (2.4) 0 Dysgeusia 2 (2.4) 0 Flushing 2 (2.4) 0 Muscle spasms 2 (2.4) 0 Pruritus 2 (2.4) 2 (5.3) Tachycardia § 2 (2.4) 0 Urticaria ¶ 2 (2.4) 0 Additional adverse reactions reported in at least 2% of patients treated with OPFOLDA in combination with Pombiliti across the 3 clinical trials i…

👥 Use in Specific Populations ~3 min read ▾

8 USE IN SPECIFIC POPULATIONS Lactation: Breastfeeding not recommended. (8.2)

8.1Pregnancy Risk Summary Based on findings from animal reproduction studies, OPFOLDA in combination with Pombiliti may cause embryo-fetal harm when administered to a pregnant female and is contraindicated during pregnancy. In a rabbit embryo-fetal development study, great vessel and cardiac malformations were increased in offspring of pregnant rabbits treated with miglustat in combination with cipaglucosidase alfa-atga at 3-fold and 16-fold, respectively, the MRHD of OPFOLDA and Pombiliti based on plasma AUC exposure.

A No Observed Adverse Effect Level (NOAEL) was not identified for the combination. In a pre- and post-natal development study in rats, increases in pup mortality were seen following maternal treatment with miglustat in combination with cipaglucosidase alfa-atga (400 mg/kg), or with cipaglucosidase alfa-atga (400 mg/kg) alone. The NOAEL for cipaglucosidase alfa-atga alone is 150 mg/kg (5-fold the Pombiliti MRHD margin).

A NOAEL for the combination was not identified. Margins at the lowest observed adverse effect level (LOAEL), relative to exposures at the MRHD of OPFOLDA and Pombiliti were 4-fold and 20-fold, respectively, based on plasma AUC exposure ( see Data ). There are no available human data on OPFOLDA in combination with Pombiliti use in pregnant females to evaluate for a drug-associated risk of major birth defects, miscarriage, or other adverse maternal or fetal outcomes.

Data Animal Data Reproductive toxicity studies of cipaglucosidase alfa-atga in rats and rabbits included pretreatment with diphenhydramine (DPH) to prevent or minimize hypersensitivity reactions. In a rabbit embryo-fetal development study, 25 mg/kg oral miglustat alone, or in combination with intravenous cipaglucosidase alfa-atga 175 mg/kg, was administered every other day to pregnant females during organogenesis (Gestation Day [GD] 7 through GD 19). Additional experimental groups received cipaglucosidase alfa-atga (30, 70, or 175 mg/kg) with the same dosing frequency during organogenesis.

Clusters of great vessel and cardiac malformations were increased in offspring of pregnant rabbits treated with the combination of miglustat and cipaglucosidase alfa-atga at 3-fold and 16-fold the MRHD of OPFOLDA and Pombiliti, respectively, based on plasma AUC exposure. A NOAEL for the combination was not identified. One fetus treated with miglustat alone (25 mg/kg) and one fetus treated with cipaglucosidase alfa-atga alone (175 mg/kg), each showed a similar cluster of these great vessel and cardiac malformations.

In a rat embryo-fetal development study, 60 mg/kg oral miglustat alone, or in combination with intravenous cipaglucosidase alfa-atga 400 mg/kg, was administered every other day to pregnant rats during organogenesis (GD 6 through GD 18). Additional experimental groups received cipaglucosidase alfa-atga (75, 150, or 400 mg/kg) with the same dosing frequency during organogenesis. No evidence of adverse effects was noted in pregnant rats or their offspring in any experimental group.

The margin at the NOAEL for miglustat (60 mg/kg) was 4-fold the OPFOLDA MRHD based on plasma AUC exposure. The margin at the NOAEL for cipaglucosidase alfa-atga (400 mg/kg) was 20-fold the Pombiliti MRHD based on plasma AUC exposure. In a pre-and post-natal development study in rats, 60 mg/kg oral miglustat alone, or in combination with intravenous cipaglucosidase alfa-atga 400 mg/kg, was administered to pregnant females every other day from GD 6 through GD 18, and from Lactation Day (LD) 1 through LD 19.

Additional experimental groups received cipaglucosidase alfa-atga (75, 150, or 400 mg/kg) with the same dosing frequency during pregnancy and lactation. Maternal and pup mortality were increased with the combination, and pup mortality was also increased with cipaglucosidase alfa-atga 400 mg/kg alone. The NOAEL for cipaglucosidase alfa-atga alone is 150 mg/kg (5-fold the Pombiliti MRHD ma…

🤰 Pregnancy ~3 min read ▾

8.1Pregnancy Risk Summary Based on findings from animal reproduction studies, OPFOLDA in combination with Pombiliti may cause embryo-fetal harm when administered to a pregnant female and is contraindicated during pregnancy. In a rabbit embryo-fetal development study, great vessel and cardiac malformations were increased in offspring of pregnant rabbits treated with miglustat in combination with cipaglucosidase alfa-atga at 3-fold and 16-fold, respectively, the MRHD of OPFOLDA and Pombiliti based on plasma AUC exposure.

A No Observed Adverse Effect Level (NOAEL) was not identified for the combination. In a pre- and post-natal development study in rats, increases in pup mortality were seen following maternal treatment with miglustat in combination with cipaglucosidase alfa-atga (400 mg/kg), or with cipaglucosidase alfa-atga (400 mg/kg) alone. The NOAEL for cipaglucosidase alfa-atga alone is 150 mg/kg (5-fold the Pombiliti MRHD margin).

A NOAEL for the combination was not identified. Margins at the lowest observed adverse effect level (LOAEL), relative to exposures at the MRHD of OPFOLDA and Pombiliti were 4-fold and 20-fold, respectively, based on plasma AUC exposure ( see Data ). There are no available human data on OPFOLDA in combination with Pombiliti use in pregnant females to evaluate for a drug-associated risk of major birth defects, miscarriage, or other adverse maternal or fetal outcomes.

Data Animal Data Reproductive toxicity studies of cipaglucosidase alfa-atga in rats and rabbits included pretreatment with diphenhydramine (DPH) to prevent or minimize hypersensitivity reactions. In a rabbit embryo-fetal development study, 25 mg/kg oral miglustat alone, or in combination with intravenous cipaglucosidase alfa-atga 175 mg/kg, was administered every other day to pregnant females during organogenesis (Gestation Day [GD] 7 through GD 19). Additional experimental groups received cipaglucosidase alfa-atga (30, 70, or 175 mg/kg) with the same dosing frequency during organogenesis.

Clusters of great vessel and cardiac malformations were increased in offspring of pregnant rabbits treated with the combination of miglustat and cipaglucosidase alfa-atga at 3-fold and 16-fold the MRHD of OPFOLDA and Pombiliti, respectively, based on plasma AUC exposure. A NOAEL for the combination was not identified. One fetus treated with miglustat alone (25 mg/kg) and one fetus treated with cipaglucosidase alfa-atga alone (175 mg/kg), each showed a similar cluster of these great vessel and cardiac malformations.

In a rat embryo-fetal development study, 60 mg/kg oral miglustat alone, or in combination with intravenous cipaglucosidase alfa-atga 400 mg/kg, was administered every other day to pregnant rats during organogenesis (GD 6 through GD 18). Additional experimental groups received cipaglucosidase alfa-atga (75, 150, or 400 mg/kg) with the same dosing frequency during organogenesis. No evidence of adverse effects was noted in pregnant rats or their offspring in any experimental group.

The margin at the NOAEL for miglustat (60 mg/kg) was 4-fold the OPFOLDA MRHD based on plasma AUC exposure. The margin at the NOAEL for cipaglucosidase alfa-atga (400 mg/kg) was 20-fold the Pombiliti MRHD based on plasma AUC exposure. In a pre-and post-natal development study in rats, 60 mg/kg oral miglustat alone, or in combination with intravenous cipaglucosidase alfa-atga 400 mg/kg, was administered to pregnant females every other day from GD 6 through GD 18, and from Lactation Day (LD) 1 through LD 19.

Additional experimental groups received cipaglucosidase alfa-atga (75, 150, or 400 mg/kg) with the same dosing frequency during pregnancy and lactation. Maternal and pup mortality were increased with the combination, and pup mortality was also increased with cipaglucosidase alfa-atga 400 mg/kg alone. The NOAEL for cipaglucosidase alfa-atga alone is 150 mg/kg (5-fold the Pombiliti MRHD margin).

A NOAEL was not identified for the combination, for which LOAEL margins…

🧒 Pediatric Use 23 words ▾

8.4Pediatric Use Safety and effectiveness of OPFOLDA in combination with Pombiliti have not been established in pediatric patients with late-onset Pompe disease.

🧓 Geriatric Use 76 words ▾

8.5Geriatric Use Of the total number of patients treated with OPFOLDA in combination with Pombiliti in clinical trials for LOPD, 17 (11%) were 65 to 74 years of age, and none were 75 years of age and older [see Clinical Studies (14) ] . Clinical trials of OPFOLDA in combination with Pombiliti did not include sufficient numbers of patients 65 years of age and older to determine whether they respond differently from younger adult patients.

🧬 Clinical Pharmacology ~2 min read ▾

12 CLINICAL PHARMACOLOGY

12.1Mechanism of Action Miglustat binds with, stabilizes, and reduces inactivation of cipaglucosidase alfa-atga in the blood after infusion. The bound miglustat is dissociated from cipaglucosidase alfa-atga after it is internalized and transported into lysosomes. Miglustat alone has no pharmacological activity in cleaving glycogen.

12.2Pharmacodynamics The exposure-response relationship and time course of pharmacodynamic response for the safety and effectiveness of miglustat have not been fully characterized.

12.3Pharmacokinetics Miglustat maximum concentration (C max ) and area under the plasma concentration-time curve (AUC) increases proportionally over a dosage range from 130 mg to 260 mg (0.5 to 1 times the approved recommended dosage of 260 mg in patients weighing ≥50 kg). At the recommended 260 mg dose, the mean (SD) C max was approximately 3 (0.9) mcg/mL and the mean AUC was approximately 25 (6.5) mcg∗hr/mL. Absorption The mean time to reach the maximum concentration (T max ) of miglustat ranged from 2 hours to 3 hours.

Effect of Food Co-administration of miglustat with food is predicted to result in delayed absorption and decreased C max in healthy subjects [see Dosage and Administration (2.2) ] . Distribution The apparent volume of distribution of miglustat was approximately 94 L in adult patients with LOPD. Elimination The apparent clearance of miglustat was approximately 10 L/hr.

The terminal elimination half‑life was approximately 6 hours. Specific Populations No clinically significant differences in the pharmacokinetics of miglustat were observed based on age (18 to 74 years) and sex. The effect of hepatic impairment on the pharmacokinetics of miglustat is unknown.

Patients with Renal Impairment The AUC 0-24hr of miglustat increased by 21%, 32%, and 41% in patients with mild (CLcr 60 to 89 mL/minute, estimated by Cockcroft-Gault), moderate (CLcr 30 to 59 mL/minute), and severe (CLcr 15 to 29 mL/minute) renal impairment, respectively, compared to patients with normal renal function. The effect of end stage renal disease on the pharmacokinetics of miglustat is unknown. Drug Interaction Studies In Vitro Studies Cytochrome P450 (CYP) Enzymes: Miglustat is not a known substrate or inhibitor of CYP1A2, CYP2A6, CYP2C9, CYP2C19, CYP2D6, CYP2E1, CYP3A4, or CYP4A11.

Transporter Systems: Miglustat is a substrate of P-gp, OCT1, and OCT2 but not a substrate of OAT1, OAT3, OATP1B1, OATP1B3, MATE1, MATE2-K, BCRP, or BSEP. Miglustat is an inhibitor of MATE1, but is not an inhibitor of OAT1, OAT3, OCT1, OCT2, OATP1B1, OATP1B3, MATE2-K, P-gp, BCRP, or BSEP.

🧬 Mechanism of Action 44 words ▾

12.1Mechanism of Action Miglustat binds with, stabilizes, and reduces inactivation of cipaglucosidase alfa-atga in the blood after infusion. The bound miglustat is dissociated from cipaglucosidase alfa-atga after it is internalized and transported into lysosomes. Miglustat alone has no pharmacological activity in cleaving glycogen.

📦 How Supplied / Storage and Handling 104 words ▾

16 HOW SUPPLIED/STORAGE AND HANDLING How Supplied OPFOLDA (miglustat) capsules are supplied as 65 mg, white opaque hard gelatin capsules with a grey opaque cap with “AT2221” printed in black on the body, and are available in bottles with child resistant caps. See Table 5 for the available OPFOLDA packages. Table 5.

OPFOLDA Packages Package Size NDC 4 count bottle 71904-300-01 24 count bottle 71904-300-02 100 count bottle 71904-300-03 Storage and Handling Store at 20°C to 25°C (68°F to 77°F). Excursions are permitted between 15°C to 30°C (59°F to 86°F), [see USP Controlled Room Temperature]. Store in the original container to protect from light.

📋 Description 116 words ▾

11 DESCRIPTION Miglustat is an N-alkylated iminosugar, a synthetic analog of D‑glucose. The pharmacologic class is enzyme stabilizer. The chemical name is 1,5-(butylimino)-1,5-dideoxy-D-glucitol with the molecular weight of 219.28 g/mol, the molecular formula C 10 H 21 NO 4 , and the following chemical structure: Miglustat is a white to off-white crystalline solid (powder).

It is highly soluble in water (>1,000 mg/mL as a free base). OPFOLDA (miglustat) capsules each contain 65 mg of miglustat for oral administration. The inactive ingredients are: colloidal silicon dioxide, magnesium stearate, microcrystalline cellulose, pregelatinized maize starch, and sucralose.

The capsule shells include black iron oxide, gelatin, and titanium dioxide. Edible printing ink includes black iron oxide, and shellac. Chemical Structure

💬 Information for Patients ~1 min read ▾

17 PATIENT COUNSELING INFORMATION Advise the patient to read the FDA‑approved patient labeling (Patient Information). OPFOLDA must be administered in combination with Pombiliti. Refer to the Pombiliti Prescribing Information for Pombiliti patient counseling information.

Administration Advise the patient and caregiver to follow the timeline recommendations for taking OPFOLDA prior to the intravenous infusion with Pombiliti and to follow the fasting recommendation. Advise the patient and caregiver that OPFOLDA should be swallowed only with unsweetened beverages [see Dosage and Administration (2.2) ] . Embryo-Fetal Toxicity OPFOLDA in combination with Pombiliti may cause embryo-fetal harm.

Advise a female patient and caregiver to inform their healthcare provider of a known or suspected pregnancy [see Warnings and Precautions (5.1) and Use in Specific Populations (8.1) ] . Advise a female of reproductive potential to use effective contraception during treatment with OPFOLDA in combination with Pombiliti and for at least 60 days after the last dose [see Use in Specific Populations (8.1, 8.3) ] . Lactation Advise a lactating female not to breastfeed during treatment with OPFOLDA in combination with Pombiliti [see Use in Specific Populations (8.2) ] .

Infertility Advise the male or female of reproductive potential that OPFOLDA in combination with Pombiliti may impair fertility [see Use in Specific Populations (8.3) ] . Manufactured for: Amicus Therapeutics US, LLC 3675 Market Street Philadelphia, PA 19104 OPFOLDA and Pombiliti are registered trademarks of Amicus Therapeutics, Inc. For more information, go to pombilitiopfoldahcp.com.

Source: FDA Structured Product Labeling, mirrored from DailyMed / openFDA. Prefer the government’s original formatting? View this label on DailyMed ↗
← 71904-0200-01 · AMICUS 71906-0080-42 · Essendant →
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.