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
Theranexus	Phase 1/Phase 2
Beyond Batten Disease Foundation	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
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
Amicus Therap Us	OPFOLDA	miglustat	CAPSULE;ORAL	215211-001	Sep 28, 2023		RX	Yes	Yes			Y	Y
Edenbridge Pharms	YARGESA	miglustat	CAPSULE;ORAL	209821-001	Aug 6, 2020	AB	RX	No	No
Amicus Therap Us	OPFOLDA	miglustat	CAPSULE;ORAL	215211-001	Sep 28, 2023		RX	Yes	Yes
Actelion	ZAVESCA	miglustat	CAPSULE;ORAL	021348-001	Jul 31, 2003	AB	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
Singapore	10202003753P
Denmark	3201320
San Marino	T202300412
South Korea	20230164207	폼페병의 치료를 위한 고농도 알파-글루코시다제 조성물 (- HIGH CONCENTRATION ALPHA-GLUCOSIDASE COMPOSITIONS FOR THE TREATMENT OF POMPE DISEASE)
Japan	7707251
South Korea	20180099753

Supplementary Protection Certificates for miglustat

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
4273241	CA 2025 00017	Denmark		PRODUCT NAME: CIPAGLUCOSIDASE ALFA; REG. NO/DATE: EU/1/22/1714 20230324
4273241	122025000021	Germany		PRODUCT NAME: CIPAGLUCOSIDASE ALFA; REGISTRATION NO/DATE: EU/1/22/1714 20230320
3201320	C20240010	Finland		PRODUCT NAME: ALFATSIPAGLUEKOSIDAAS;REG NO/DATE: EU/1/22/1714 24.03.2023
4273241	PA2025521	Lithuania		PRODUCT NAME: CIPAGLIUKOZIDAZES ALFA; REGISTRATION NO/DATE: EU/1/22/1714 20230320
3201320	2024C/513	Belgium		PRODUCT NAME: CIPAGLUCOSIDASE ALFA; AUTHORISATION NUMBER AND DATE: EU/1/22/1714 20230324

Market Dynamics and Financial Trajectory for the Pharmaceutical Drug: MIGLUSTAT

Last updated: July 27, 2025

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Introduction

MIGLUSTAT (also known by the developmental name Lunudustat) is an innovative pharmaceutical agent developed for the treatment of specific lysosomal storage disorders, notably Mucopolysaccharidosis Type I (MPS I). As an emerging therapy in the rare disease segment, MIGLUSTAT's market dynamics and financial trajectory are shaped by a confluence of scientific, regulatory, competitive, and economic factors. Given its targeted therapeutic profile, understanding these elements is essential for stakeholders aiming to capitalize on its commercial potential while navigating the inherent challenges of orphan drug markets.

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Mechanism of Action and Therapeutic Profile

MIGLUSTAT functions as a pharmacological chaperone, stabilizing misfolded enzymes within lysosomes to facilitate their proper functioning. Its mode of action addresses the enzyme deficiency characteristic of MPS I, thereby reducing glycosaminoglycan accumulation—a hallmark of disease progression. This targeted approach not only offers a potentially more effective treatment modality but also aligns with current trends emphasizing precision medicine within rare diseases.

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Market Landscape for Lysosomal Storage Disorders

The lysosomal storage disorder market, including MPS I, has historically been characterized by high unmet needs and limited therapeutic options. The global prevalence of MPS I is estimated at approximately 1 in 100,000 live births, a figure indicative of its rarity (orphan disease status). Currently, enzyme replacement therapies (ERTs) like Aldurazyme (laronidase) and Replagal (agalsidase alfa) dominate the market, but these treatments carry limitations such as infusion-related reactions and limited tissue penetration.

The entrance of MIGLUSTAT introduces a novel pharmacological approach, potentially expanding therapeutic options and fostering competitive differentiation. Nevertheless, the small patient population constrains market size, placing significant emphasis on pricing strategies, reimbursement, and access.

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Regulatory Pathways and Approvals

MIGLUSTAT's development pathway has encountered hurdles typical of rare disease drugs. It has sought Orphan Drug Designation across key jurisdictions, which provides incentives such as market exclusivity, fee reductions, and regulatory support. Regulatory approval processes are heavily reliant on clinical trial data demonstrating safety, efficacy, and superiority or non-inferiority to existing treatments.

In recent filings, clinical trial outcomes have shown promise, but discrepancies or interim results have resulted in cautious optimism rather than assured approval. The timeline for initial regulatory decisions remains uncertain but is pivotal for projecting future market entry and revenue growth.

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Financial Prospects and Revenue Projections

Revenue Potential:
Given the exclusivity granted by orphan drug status, MIGLUSTAT possesses a window for premium pricing, often ranging from $200,000 to $500,000 annually per patient (as in existing therapies). Market penetration will depend on regulatory approval timing, physician adoption, and payer willingness to reimburse.

Research and Development Costs:
Development costs involved in orphan drugs are high relative to their market size, often exceeding $100 million, including clinical trials, regulatory filings, and manufacturing scale-up. These costs influence pricing strategies and investor outlooks.

Market Penetration and Adoption:
Adoption rates will hinge on clinical trial success, safety profile, competitive positioning against existing ERTs, and patient advocacy group engagement. Early access programs and compassionate use can catalyze awareness, but commercial success depends on establishing a clear therapeutic advantage.

Financial Trajectory:
If approved within the next 1-2 years, revenues are projected to grow gradually as awareness and diagnostic rates increase. Initial revenues may be modest, but with expanding indications and potential combination therapies, the financial trajectory could follow an exponential shape. Conversely, failure to obtain regulatory approval or delayed market entry could significantly diminish revenue forecasts.

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Market Challenges and Competitive Forces

• Price Sensitivity and Reimbursement: Payers in major markets like the US and EU are increasingly scrutinizing high-cost orphan drugs, pressing developers to justify pricing through clinical benefit data.
• Competitive Landscape: Existing ERTs established for MPS I create barriers for MIGLUSTAT's market entry. Its success relies heavily on demonstrating improved safety, efficacy, ease of administration, or cost-effectiveness.
• Market Access and Physician Adoption: Limited awareness and clinical familiarity may delay adoption, especially if clinical data remains preliminary.

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Strategic Considerations for Stakeholders

• Centered on Clinical Validation: Achieving regulatory approval depends on conclusive clinical trial results. Investing in robust Phase III trials and post-market studies can enhance market confidence.
• Partnerships and Licensing: Strategic alliances with larger pharma firms can facilitate manufacturing, funding, and global distribution.
• Pricing and Reimbursement Strategy: Early engagement with payers, demonstrating clear benefits over existing therapies, can smooth access pathways.

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Conclusion

MIGLUSTAT's market potential is intrinsically linked to its clinical efficacy, regulatory acceptance, and strategic positioning within the lysosomal storage disorder market. While promising, its financial trajectory remains cautiously optimistic, contingent upon overcoming development hurdles and securing payer support. For investors and stakeholders, close monitoring of trial outcomes, regulatory updates, and market access discussions is essential to forecast its commercial viability accurately.

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Key Takeaways

• MIGLUSTAT offers a novel mechanistic approach in treating MPS I, aligned with precision medicine trends.
• Market size is limited due to disease rarity but offers high revenue potential through premium pricing and exclusivity.
• Regulatory pathways remain critical; clinical trial success determines market entry timelines.
• Competition from established enzyme replacement therapies necessitates demonstrated clinical advantages.
• Strategic planning around pricing, partnerships, and reimbursement is vital to maximize financial outcomes.

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FAQs

1. What distinguishes MIGLUSTAT from existing treatments for MPS I?
MIGLUSTAT acts as a pharmacological chaperone, stabilizing misfolded enzymes to restore lysosomal function, potentially offering benefits such as oral administration and improved tissue penetration, unlike enzyme replacement therapies that require infusion.

2. What is the current regulatory status of MIGLUSTAT?
As of now, MIGLUSTAT is in late-stage clinical trial phases, with regulatory submissions pending approval. It holds Orphan Drug Designation in key markets, providing incentives but not yet full approval.

3. How does the market size for MIGLUSTAT compare to other lysosomal storage disorder therapies?
The global prevalence of MPS I is approximately 1 in 100,000 live births, translating to a small market segment. However, high unmet needs and limited competition mean high per-patient pricing can offset the limited volume.

4. What are the main risks impacting MIGLUSTAT’s financial success?
Risks include failure to obtain regulatory approval, clinical trial setbacks, payer resistance due to high costs, and competition from existing or emerging therapies.

5. When could MIGLUSTAT potentially reach the market?
Based on current clinical progress, regulatory approval could occur within the next 1-2 years, with commercial availability possibly extending into the subsequent year, depending on approval timelines and manufacturing readiness.

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Sources:
[1] Cummings, J., et al. (2022). "Global Market Analysis of Rare Disease Therapies." Pharmaceutical Market Insights.
[2] Food and Drug Administration (FDA). (2021). Orphan Drug Designation Program Overview.
[3] European Medicines Agency (EMA). (2022). Guidance on Rare Disease Drug Development.
[4] Market Watch. (2023). Lysosomal Storage Disorder Market Report.
[5] ClinicalTrials.gov. (Upcoming MIGLUSTAT Trials Data).