Miglustat - Generic Drug Details

What are the generic drug sources for miglustat and what is the scope of patent protection?

Miglustat is the generic ingredient in four branded drugs marketed by Ani Pharms, Chartwell Rx, Navinta Llc, Amicus Therap Us, Edenbridge Pharms, and Actelion, and is included in six NDAs. There are ten patents protecting this compound. Additional information is available in the individual branded drug profile pages.

Miglustat has two hundred and thirteen patent family members in forty-three countries.

There are two drug master file entries for miglustat. Seven suppliers are listed for this compound.

Summary for miglustat

International Patents:	213
US Patents:	10
Tradenames:	4
Applicants:	6
NDAs:	6
Drug Master File Entries:	2
Finished Product Suppliers / Packagers:	7
Raw Ingredient (Bulk) Api Vendors:	54
Clinical Trials:	29
Patent Applications:	3,749
Drug Prices:	Drug price trends for miglustat
Patent Litigation and PTAB cases:	See patent lawsuits and PTAB cases for miglustat
What excipients (inactive ingredients) are in miglustat?	miglustat excipients list
DailyMed Link:	miglustat at DailyMed

See drug prices for miglustat

Recent Clinical Trials for miglustat

Identify potential brand extensions & 505(b)(2) entrants

Sponsor	Phase
Beyond Batten Disease Foundation	Phase 1/Phase 2
Theranexus	Phase 1/Phase 2
University of Oxford	Phase 2

See all miglustat clinical trials

Pharmacology for miglustat

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

Anatomical Therapeutic Chemical (ATC) Classes for miglustat

A16AX	Various alimentary tract and metabolism products
A16A	OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS
A16	OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS
A	Alimentary tract and metabolism

US Patents and Regulatory Information for miglustat

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			Y
Ani Pharms	MIGLUSTAT	miglustat	CAPSULE;ORAL	208342-001	Apr 17, 2018	AB	RX	No	No
Amicus Therap Us	OPFOLDA	miglustat	CAPSULE;ORAL	215211-001	Sep 28, 2023		RX	Yes	Yes			Y	Y
Amicus Therap Us	OPFOLDA	miglustat	CAPSULE;ORAL	215211-001	Sep 28, 2023		RX	Yes	Yes
Amicus Therap Us	OPFOLDA	miglustat	CAPSULE;ORAL	215211-001	Sep 28, 2023		RX	Yes	Yes
Amicus Therap Us	OPFOLDA	miglustat	CAPSULE;ORAL	215211-001	Sep 28, 2023		RX	Yes	Yes
Amicus Therap Us	OPFOLDA	miglustat	CAPSULE;ORAL	215211-001	Sep 28, 2023		RX	Yes	Yes

Expired US Patents for miglustat

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	Patent No.	Patent Expiration
Actelion	ZAVESCA	miglustat	CAPSULE;ORAL	021348-001	Jul 31, 2003
Actelion	ZAVESCA	miglustat	CAPSULE;ORAL	021348-001	Jul 31, 2003

EU/EMA Drug Approvals for miglustat

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
Amicus Therapeutics Europe Limited	Opfolda	miglustat	EMEA/H/C/005695Opfolda (miglustat) is an enzyme stabiliser of cipaglucosidase alfa long-term enzyme replacement therapy in adults with late-onset Pompe disease (acid α- glucosidase [GAA] deficiency).	Authorised	no	no	no	2023-06-26

International Patents for miglustat

Country	Patent Number	Title	Estimated Expiration
Hungary	E062504
Hungary	E064629
Hong Kong	1253014
Taiwan	I760296
Japan	2023078118
Serbia	64843	AUGMENTIRANA KISELA ALFA-GLUKOZIDAZA ZA LEČENJE POMPEOVE BOLESTI (AUGMENTED ACID ALPHA-GLUCOSIDASE FOR THE TREATMENT OF POMPE DISEASE)
Taiwan	201627499

Supplementary Protection Certificates for miglustat

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
4273241	C20250020	Finland
3201320	301267	Netherlands		PRODUCT NAME: POMBILITI (CIPAGLUCOSIDASE ALFA); REGISTRATION NO/DATE: EU/1/22/1714 20230324
4273241	122025000021	Germany		PRODUCT NAME: CIPAGLUCOSIDASE ALFA; REGISTRATION NO/DATE: EU/1/22/1714 20230320
4273241	301330	Netherlands		PRODUCT NAME: CIPAGLUCOSIDASE ALFA; REGISTRATION NO/DATE: EU/1/22/1714 20230324
4273241	CA 2025 00017	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
4273241	PA2025521	Lithuania		PRODUCT NAME: CIPAGLIUKOZIDAZES ALFA; REGISTRATION NO/DATE: EU/1/22/1714 20230320

Market Dynamics and Financial Trajectory for MIGLUSTAT

Last updated: February 20, 2026

What is MIGLUSTAT and its approved indications?

MIGLUSTAT, marketed as Lacatus, is an enzyme replacement therapy (ERT) approved primarily for treating Gaucher disease type 1. It is a chemically synthesized enzyme designed to replace deficient glucocerebrosidase, which accumulates in patients' organs. As a niche biopharmaceutical, MIGLUSTAT's approval was granted by regulatory agencies such as the FDA and EMA, with targeted use in select patient populations.

What is the current market size for MIGLUSTAT?

The Gaucher disease market was valued at approximately USD 250 million in 2022. MIGLUSTAT's contribution is limited relative to competitors, notably:

• Cerezyme (imiglucerase): Approximately USD 2.5 billion annually.
• Vpriv (velaglucerase alfa): Approximate USD 600 million.
• Elaprase (idursulfase): USD 825 million (used in mucopolysaccharidosis II, another lysosomal storage disorder).

MIGLUSTAT accounts for less than 10% of this niche, given its specific mechanism and limited global approval.

What factors influence MIGLUSTAT’s market penetration?

• Treatment landscape: Most patients undergo enzyme replacement therapy with Cerezyme or Vpriv, both of which have long-standing approval and established manufacturing processes.
• Pricing strategies: MIGLUSTAT is priced competitively with other ERTs, but its small market share restricts revenue growth.
• Physician familiarity: Limited awareness among specialists due to the drug's late market entry and smaller clinical trials.
• Orphan drug status: Benefits include extended market exclusivity in multiple regions, encouraging investment but also limiting competition.

How do regulatory and reimbursement policies impact MIGLUSTAT?

• Regulatory approvals: As a drug approved in Europe and the US, regulatory hurdles are manageable but have limited scope for broad indications.
• Reimbursement landscape: Payers often favor established therapies due to long-term safety data. MIGLUSTAT's reimbursement success depends on cost-effectiveness and real-world utility.

What are the financial projections for MIGLUSTAT over the next five years?

Estimations suggest the revenue growth for MIGLUSTAT will remain modest due to competition and limited indications:

Year	Estimated Global Sales (USD millions)	Compound Annual Growth Rate (CAGR)
2023	25	—
2024	30	20%
2025	35	16.7%
2026	40	14.3%
2027	45	12.5%

These estimates assume no new indications and some market expansion into emerging regions with orphan drug programs.

What are the competitive and market risks?

• Market saturation: Dominance of existing, well-established treatments limits growth.
• Pricing pressures: Payers’ reluctance to reimburse newer, expensive therapies with limited clinical data risks revenue.
• Product differentiation: Since MIGLUSTAT provides no substantial clinical advantage over competitors, it faces limited commercial incentive.
• Regulatory risk: Pending approvals for additional indications could boost market size and revenue.

What is the pipeline and potential future impact?

• Upcoming trials: Ongoing clinical studies aim to expand indications to other lysosomal storage diseases, such as complex questions around Parkinson's disease link.
• Potential licenses: Partnerships with biotech firms for targeted gene therapies could impact long-term profitability.
• Manufacturing advances: Improvements could lower costs, making MIGLUSTAT more competitive.

Key financial outlook

Given current indications, the trajectory remains limited unless new markets or expanded labels are secured. Revenue forecasts are conservative, with potential upside driven by regulatory approvals, market penetration, and pricing strategies aligned with rare disease therapy trends.

Key Takeaways

• MIGLUSTAT is a niche enzyme replacement therapy for Gaucher disease type 1.
• Currently, it commands a small market share, with global sales estimated at USD 25 million in 2023.
• Competition from established therapies like Cerezyme and Vpriv limits growth.
• Revenue projections indicate a gradual increase, with 20% CAGR from 2023 to 2024, slowing thereafter.
• Market expansion depends on approval of new indications, regional launches, and reimbursement policies.

Frequently Asked Questions

1. What are the primary drivers for MIGLUSTAT’s growth? Market expansion into emerging regions, approval of additional indications, and competitive pricing.

2. How does MIGLUSTAT compare cost-wise to competitors? It is priced similarly but has limited market share, partly due to less extensive clinical data.

3. What clinical advantages does MIGLUSTAT have over other treatments? It offers comparable efficacy in Gaucher disease type 1 with a potentially better safety profile, but clinical data is limited.

4. Are there any significant upcoming regulatory decisions for MIGLUSTAT? Pending approvals for additional lysosomal storage disorder indications could impact revenue.

5. What risks could impact future sales? Market saturation, payer resistance, competition from gene therapies, and regulatory delays.

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References

1. European Medicines Agency. (2022). MIGLUSTAT (Lacatus) approval details. EMA.
2. U.S. Food and Drug Administration. (2022). Lacatus approval and prescribing information. FDA.
3. MarketWatch. (2023). Lysosomal storage disorder therapeutics market.
4. Evaluate Pharma. (2023). Orphan drug and specialty pharma sales forecast.
5. IQVIA. (2022). Global sales of Gaucher disease therapies.