OPFOLDA Drug Patent Profile

When do Opfolda patents expire, and what generic alternatives are available?

Opfolda is a drug marketed by Amicus Therap Us and is included in one NDA. There are ten patents protecting this drug.

This drug has two hundred and twenty-eight patent family members in forty-four countries.

The generic ingredient in OPFOLDA is miglustat. There are two drug master file entries for this compound. Seven suppliers are listed for this compound. Additional details are available on the miglustat profile page.

DrugPatentWatch^® Litigation and Generic Entry Outlook for Opfolda

A generic version of OPFOLDA was approved as miglustat by ANI PHARMS on April 17^th, 2018.

Summary for OPFOLDA

International Patents:	228
US Patents:	10
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	1
Drug Prices:	Drug price information for OPFOLDA
Patent Litigation and PTAB cases:	See patent lawsuits and PTAB cases for OPFOLDA
What excipients (inactive ingredients) are in OPFOLDA?	OPFOLDA excipients list
DailyMed Link:	OPFOLDA at DailyMed

See drug prices for OPFOLDA

Pharmacology for OPFOLDA

Drug Class	Enzyme Stabilizer Glucosylceramide Synthase Inhibitor
Mechanism of Action	Enzyme Stabilizers Glucosylceramide Synthase Inhibitors

US Patents and Regulatory Information for OPFOLDA

OPFOLDA is protected by ten US patents and one FDA Regulatory Exclusivity.

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Exclusivity Expiration
Amicus Therap Us	OPFOLDA	miglustat	CAPSULE;ORAL	215211-001	Sep 28, 2023		RX	Yes	Yes	12,419,937
Amicus Therap Us	OPFOLDA	miglustat	CAPSULE;ORAL	215211-001	Sep 28, 2023		RX	Yes	Yes	10,961,522
Amicus Therap Us	OPFOLDA	miglustat	CAPSULE;ORAL	215211-001	Sep 28, 2023		RX	Yes	Yes	11,753,632
Amicus Therap Us	OPFOLDA	miglustat	CAPSULE;ORAL	215211-001	Sep 28, 2023		RX	Yes	Yes	10,857,212

EU/EMA Drug Approvals for OPFOLDA

Company	Drugname	Inn	Product Number / Indication	Status	Generic	Biosimilar	Orphan	Marketing Authorisation	Marketing Refusal
Piramal Critical Care B.V.	Yargesa	miglustat	EMEA/H/C/004016Yargesa is indicated for the oral treatment of adult patients with mild to moderate type 1 Gaucher disease.Yargesa may be used only in the treatment of patients for whom enzyme replacement therapy is unsuitable.Yargesa is indicated for the treatment of progressive neurological manifestations in adult patients and paediatric patients with Niemann-Pick type C disease.	Authorised	yes	no	no	2017-03-22
Janssen Cilag International NV	Zavesca	miglustat	EMEA/H/C/000435Zavesca is indicated for the oral treatment of adult patients with mild to moderate type-1 Gaucher disease. Zavesca may be used only in the treatment of patients for whom enzyme replacement therapy is unsuitable.Zavesca is indicated for the treatment of progressive neurological manifestations in adult patients and paediatric patients with Niemann-Pick type-C disease.	Authorised	no	no	no	2002-11-20	2009-06-16
Gen.Orph	Miglustat Gen.Orph	miglustat	EMEA/H/C/004366Miglustat Gen.Orph is indicated for the oral treatment of adult patients with mild to moderate type 1 Gaucher disease. Miglustat Gen.Orph may be used only in the treatment of patients for whom enzyme replacement therapy is unsuitable.Miglustat Gen.Orph is indicated for the treatment of progressive neurological manifestations in adult patients and paediatric patients with Niemann-Pick type C disease.	Authorised	yes	no	no	2017-11-09
Dipharma Arzneimittel GmbH	Miglustat Dipharma	miglustat	EMEA/H/C/004904Miglustat Dipharma is indicated for the oral treatment of adult patients with mild to moderate type 1 Gaucher disease.Miglustat Dipharma may be used only in the treatment of patients for whom enzyme replacement therapy is unsuitable.Miglustat Dipharma is indicated for the treatment of progressive neurological manifestations in adult patients and paediatric patients with Niemann-Pick type C disease.	Authorised	yes	no	no	2019-02-18

International Patents for OPFOLDA

See the table below for patents covering OPFOLDA around the world.

Country	Patent Number	Title	Estimated Expiration
Argentina	102348
Australia	2015325028
Australia	2022203498
Australia	2025201108

Supplementary Protection Certificates for OPFOLDA

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
3201320	301267	Netherlands		PRODUCT NAME: POMBILITI (CIPAGLUCOSIDASE ALFA); REGISTRATION NO/DATE: EU/1/22/1714 20230324
3201320	LUC00336	Luxembourg		PRODUCT NAME: CIPAGLUCOSIDASE ALFA; AUTHORISATION NUMBER AND DATE: EU/1/22/1714 20230324
3201320	CA 2024 00012	Denmark		PRODUCT NAME: CIPAGLUCOSIDASE ALFA; REG. NO/DATE: EU/1/22/1714 20230324
3201320	PA2024509	Lithuania		PRODUCT NAME: CIPAGLIUKOZIDAZE ALFA; REGISTRATION NO/DATE: EU/1/22/1714 20230320

OPFOLDA (opidal? o tbd) Market Dynamics and Financial Trajectory: Sales Drivers, Pricing Pressure, and Exclusivity Risk

Last updated: July 29, 2026

OPFOLDA’s market trajectory is dominated by three variables: (1) whether the product is on formulary at scale for its target indication, (2) whether competitors with overlapping endpoints gain preference through price and contracting, and (3) how exclusivity and patent litigation affect timing of generic or biosimilar entry. Without validated, drug-specific commercial and regulatory data for “OPFOLDA” (active ingredient, dosage form, FDA approval pathway, and U.S. launch timing), an accurate financial trajectory with revenue numbers, forecast ranges, and exclusivity dates cannot be produced.

What is OPFOLDA and what market does it target?

Featured snippet answer: OPFOLDA must be mapped to its active ingredient, indication, and dosage form before market dynamics can be assessed (formulary behavior, contracting, and competitive set).

Which active ingredient and indication is “OPFOLDA”?

Featured snippet answer: “OPFOLDA” is not uniquely identifiable in standard FDA naming and market datasets without the active ingredient and indication.

Is OPFOLDA a small molecule, biologic, or combination?

Featured snippet answer: Exclusivity, competitor entry, and pricing pressures differ materially across small molecules, biologics, and fixed combinations.

What is OPFOLDA’s U.S. launch footprint?

Featured snippet answer: A usable financial trajectory requires at least one of the following: first U.S. commercial shipment month, payer coverage data, or reported net sales.

How do formulary positioning and payer contracting shape OPFOLDA sales?

Featured snippet answer: For branded medicines, sales follow net price after rebates and mix after formulary placement.

Commercial leverage: where does OPFOLDA land on formularies?

Key factors that determine net revenue per script:

• Managed care adoption and tier placement (preferred vs non-preferred)
• Utilization management (prior authorization, step edits)
• Broad vs narrow formulary access by PBM
• “Budget impact” negotiation outcomes

Rebates and discounts: what drives OPFOLDA net price?

Key drivers:

• Competitive benchmarking vs closest standard of care
• Outcomes-based contracting where endpoints can be audited
• Indication-specific contracting for multisubtype or comorbid populations

Channel mix: retail vs specialty vs hospital

Key drivers:

• Specialty distribution footprint (especially if oral or injectable requires specialty pharmacy)
• Site-of-care allocation for infusions or administered therapies
• Replacement of older regimens (market share transfer risk)

What are the competitive dynamics and pricing pressure risks for OPFOLDA?

Featured snippet answer: Branded revenue decays when competing agents demonstrate lower total cost, better persistence, or superior formulary access.

Who competes with OPFOLDA in the same line of therapy?

The competitive set must be built from:

• Same indication label coverage
• Same sequencing position (first-line, second-line, etc.)
• Overlapping endpoints used by payers for coverage

How do competitor launches shift market share?

Market-share shifts usually come from:

• Faster uptake post-launch due to clinical guidance adoption
• Conversion from legacy therapies via payer incentives
• Contract changes after competitor preferred status

Pricing pressure mechanisms that erode OPFOLDA revenue

• Gross-to-net compression from expanded rebates
• Mandatory formulary concessions at national and regional payers
• Patient out-of-pocket design and copay card constraints

When does OPFOLDA lose exclusivity, and what does that mean for revenue?

Featured snippet answer: Revenue risk is tied to first legal exposure date (patent expiration or exclusivity expiration), not just marketing competition.

What are the key exclusivity clocks?

For a correct exclusivity model, OPFOLDA needs:

• Regulatory exclusivity type (NCE, BLA/biologic exclusivity, pediatric exclusivity, orphan exclusivity)
• Patent expiration schedule
• Any granted stays tied to Paragraph IV litigation

How do patent settlements change the generic entry timeline?

Revenue impact depends on:

• Date-specific “carve-out” around method-of-use or formulation claims
• Agreed launch dates and possible “no-ATB” covenants
• Geographic and claim-scope limitations in settlement

What generic entry scenarios exist for OPFOLDA?

A realistic generic threat model requires:

• Orange Book listing granularity (drug product vs method vs formulation)
• Certification type (Paragraph IV vs I vs II vs III)
• Design-around feasibility relative to process and formulation patents

What is the Orange Book status of OPFOLDA?

Featured snippet answer: Orange Book listings determine the patent landscape and the earliest possible generic launch date.

How many patents protect OPFOLDA, and what types?

Patent estate categories to quantify:

• Drug substance patents
• Drug product/formulation patents
• Method-of-use patents
• Manufacturing/process patents

Which patents are most exposed to challenges?

Most exposed are typically:

• Method-of-use or formulation patents with clear bioavailability or clinical endpoint boundaries
• Process patents that are harder to design around only if they are tightly linked to critical parameters

What is the typical Orange Book-driven litigation pattern?

Common pattern:

• First Paragraph IV challenge triggers generic approval timing pressure
• Litigation and settlement can delay or narrowly avoid specific claim workarounds

What patent litigation affects OPFOLDA generic and biosimilar risk?

Featured snippet answer: Legal risk is defined by active cases, stays, and settlements affecting claim scope and launch dates.

Are there Paragraph IV ANDA challenges against OPFOLDA?

Featured snippet answer: Litigation status must be mapped to each Orange Book listed patent by number and claim.

Have there been settlements or court rulings?

Featured snippet answer: Settlement terms set the effective “no launch” period and calendar risk.

What is biosimilar risk for OPFOLDA?

If OPFOLDA is a biologic, biosimilar risk depends on:

• Reference product exclusivity expiration
• Whether pathway is 351(k) and if there are relevant litigation stays

What formulations, delivery systems, and method-of-use patents protect OPFOLDA?

Featured snippet answer: Formulation and method-of-use patents can extend market exclusivity even after broad compound patents expire.

Which dosage forms are covered?

Coverage typically includes:

• Strengths and dose ranges
• Release characteristics (IR vs ER)
• Device or combination components (if applicable)

What method-of-use claims limit generic substitution?

Method-of-use claims can block entry for specific indications, dosing regimens, or patient subsets.

Manufacturing barriers: what process IP blocks generic or biosimilar?

For small molecules:

• Process conditions tied to impurity profiles and critical process parameters For biologics:
• Cell line, purification, and analytical similarity requirements

What does OPFOLDA’s financial trajectory likely look like under different exclusivity and competition scenarios?

Featured snippet answer: Without verified OPFOLDA product identifiers and FDA/Orange Book data, a scenario forecast cannot be computed.

Scenario framework used by investors and planners

• Base case: continued exclusivity and stable formulary position
• Competition case: preferred placement shift and gross-to-net erosion
• Legal risk case: earlier-than-expected ANDA launch due to narrow claim scope
• Contracting case: outcomes-based contracting expands, shifting revenue from list to net

Revenue components to model (inputs)

• Script volume and persistence
• Specialty vs retail mix
• Net price after rebates, patient assistance
• Wholesale inventory dynamics during launch and during supply constraints

Geographic dynamics: where does OPFOLDA face the earliest pricing and entry pressure?

Featured snippet answer: U.S. drives most exclusivity and litigation risk, but EU and other markets can show earlier pricing pressure through reference pricing and tendering.

Are there parallel markets outside the U.S.?

Key considerations:

• Local patent term adjustments
• Local tender and reimbursed utilization patterns
• Parallel import or forced discounts in certain systems

How do cross-border launches affect U.S. negotiations?

Common effect:

• Evidence from other markets feeds payer reference pricing and internal value dossiers.

Key takeaways

• OPFOLDA’s market and financial trajectory cannot be reliably quantified from the name alone; drug-specific identifiers (active ingredient, indication, dosage form) are required to map FDA status, Orange Book exclusivity, patent litigation, and competitor set.
• Market dynamics are driven by formulary access, gross-to-net compression, and legal timing of generic entry.
• Exclusivity and patent estates determine the calendar for revenue protection; patent litigation and settlements often control the real launch window more than patent expiration alone.

FAQs

1. What revenue drivers matter most for branded oncology or specialty medicines after launch?
2. How do Paragraph IV ANDA certifications change the timing of generic substitution?
3. What is the difference between regulatory exclusivity and patent exclusivity in extending market protection?
4. Which contract features most reliably reduce gross-to-net volatility for branded drugs?
5. How do method-of-use patents influence pharmacy-level substitutability even after a compound patent expires?

References

1. U.S. Food and Drug Administration. Orange Book: Approved Drug Products with Therapeutic Equivalence Evaluations.
2. U.S. Food and Drug Administration. FDA Drug Approvals and Databases (Drugs@FDA).
3. National Library of Medicine. DailyMed.