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, Breckenridge, Amicus Therap Us, Edenbridge Pharms, and Actelion, and is included in five NDAs. There are seven patents protecting this compound. Additional information is available in the individual branded drug profile pages.

Miglustat has one hundred and thirty-seven patent family members in forty-one countries.

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

Summary for miglustat

International Patents:	137
US Patents:	7
Tradenames:	4
Applicants:	5
NDAs:	5
Drug Master File Entries:	2
Finished Product Suppliers / Packagers:	5
Raw Ingredient (Bulk) Api Vendors:	64
Clinical Trials:	29
Patent Applications:	3,071
Drug Prices:	Drug price trends 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	11,278,601		Y
Amicus Therap Us	OPFOLDA	miglustat	CAPSULE;ORAL	215211-001	Sep 28, 2023		RX	Yes	Yes	11,278,599
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	10,857,212
Amicus Therap Us	OPFOLDA	miglustat	CAPSULE;ORAL	215211-001	Sep 28, 2023		RX	Yes	Yes	10,208,299		Y	Y
Actelion	ZAVESCA	miglustat	CAPSULE;ORAL	021348-001	Jul 31, 2003	AB	RX	Yes	Yes
Amicus Therap Us	OPFOLDA	miglustat	CAPSULE;ORAL	215211-001	Sep 28, 2023		RX	Yes	Yes	11,753,632

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	5,472,969
Actelion	ZAVESCA	miglustat	CAPSULE;ORAL	021348-001	Jul 31, 2003	5,525,616

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/004016 Yargesa 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/000435 Zavesca 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/004366 Miglustat 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/004904 Miglustat 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/005695 Opfolda (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
Israel	283837	חומצה אלפא-גלוקוסידאז משופרת לטיפול במחלת פומפ (Augmented acid alpha-glucosidase for the treatment of pompe disease)
Denmark	3201320
Malaysia	198085	AUGMENTED ACID ALPHA-GLUCOSIDASE FOR THE TREATMENT OF POMPE DISEASE
Chile	2018001773	Alfa-glucosidasa con mayor cantidad de ácido para el tratamiento de la enfermedad de pompe
South Korea	20230066482	폼페병의 치료를 위한 고농도 알파-글루코시다제 조성물 (- HIGH CONCENTRATION ALPHA-GLUCOSIDASE COMPOSITIONS FOR THE TREATMENT OF POMPE DISEASE)
Taiwan	I760296
El Salvador	2017005416	ALFA-GLUCOSIDASA ÁCIDA MUY POTENTE CON HIDRATOS DE CARBONO POTENCIADOS

Supplementary Protection Certificates for miglustat

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
3201320	PA2024509	Lithuania		PRODUCT NAME: CIPAGLIUKOZIDAZE 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
3201320	301267	Netherlands		PRODUCT NAME: POMBILITI (CIPAGLUCOSIDASE ALFA); REGISTRATION NO/DATE: EU/1/22/1714 20230324
3201320	CA 2024 00012	Denmark		PRODUCT NAME: CIPAGLUCOSIDASE ALFA; REG. NO/DATE: EU/1/22/1714 20230324
3201320	122024000016	Germany		PRODUCT NAME: CIPAGLUCOSIDASE ALFA; REGISTRATION NO/DATE: EU/1/22/1714 20230320
3201320	LUC00336	Luxembourg		PRODUCT NAME: CIPAGLUCOSIDASE ALFA; AUTHORISATION NUMBER AND DATE: EU/1/22/1714 20230324

Miglustat Market Analysis and Financial Projection Experimental

Market Dynamics and Financial Trajectory for Miglustat

Introduction to Miglustat

Miglustat, a small molecule drug, is primarily used in the treatment of various rare diseases, including glycogen storage disease type II, neurologic manifestations of Niemann-Pick diseases, Gaucher disease, and Niemann-Pick disease type C. Developed by Actelion Pharmaceuticals Ltd., miglustat has been approved globally since its first approval in the European Union in November 2002[4].

Market Size and Growth

The global market for miglustat API is projected to grow steadily over the coming years. As of 2023, the market was estimated to be worth US$ 195 million. It is forecasted to reach a readjusted size of US$ 252.7 million by 2030, with a Compound Annual Growth Rate (CAGR) of 3.8% during the forecast period of 2024-2030[1].

Key Players

The miglustat API market is dominated by several key players, including MYLAN LABORATORIES LTD, DIPHARMA SA, and NAVINTA LLC. These companies play a crucial role in the production, distribution, and commercialization of miglustat, influencing the market dynamics and competitive landscape[1].

Applications and Types

Miglustat is used in various applications, primarily in the form of capsules and other formulations. The drug is available in different purity levels, with the main types being Purity ≥99% and Purity <99%. These variations cater to different clinical needs and regulatory requirements[1].

Mechanism of Action

Miglustat targets the UGCG enzyme, which is involved in the biosynthesis of glucosylceramide, a key component in the pathophysiology of several lysosomal storage diseases. By inhibiting this enzyme, miglustat reduces the accumulation of toxic substances in cells, thereby alleviating the symptoms of these diseases[4].

Regulatory Status

Miglustat has undergone priority review and has been designated as an orphan drug, which provides incentives for its development and commercialization. This designation highlights its potential as a therapeutic option for rare diseases and underscores the regulatory support it has received globally[4].

Financial Performance of Key Associated Companies

Amicus Therapeutics

Amicus Therapeutics, a company closely associated with the commercialization of miglustat through its combination therapy Pombiliti + Opfolda, has shown robust financial performance. In 2023, Amicus reported a 21% increase in total revenue to $399.4 million, driven by strong patient demand for Galafold and the successful launch of Pombiliti + Opfolda. The company anticipates double-digit revenue growth for Galafold and continued strong demand for Pombiliti + Opfolda, which includes miglustat as a component[2].

Revenue Growth and Projections

• Galafold Revenue: Amicus Therapeutics projects Galafold revenue growth of 14-18% at constant exchange rates (CER) for the full year 2023, driven by underlying demand, geographic expansion, and commercial execution[5].
• Pombiliti + Opfolda: The commercial launch of Pombiliti + Opfolda is underway in key markets, with 120 patients on treatment or scheduled to be treated as of early 2023. This combination therapy, which includes miglustat, has been honored with the 2024 New Treatment Award, recognizing its potential impact on lysosomal diseases[2].

Operational Efficiency and Cost Management

Amicus Therapeutics has demonstrated a commitment to operational efficiency and cost management. The company reported a decrease in both GAAP and non-GAAP operating expenses in 2023 compared to the previous year, reflecting a disciplined approach to expense management. This efficiency is crucial for achieving non-GAAP profitability, which was achieved in the fourth quarter of 2023[2].

Market Penetration and Patient Demand

The strong patient demand for Galafold and the growing adoption of Pombiliti + Opfolda indicate a high level of market acceptance for these treatments. As of the end of 2023, over 2,400 people living with Fabry disease were on Galafold treatment, and the number of patients on Pombiliti + Opfolda is increasing steadily. This demand is a critical metric for assessing the long-term sustainability of the revenue streams from these therapies[2].

Geographic Expansion

Miglustat and associated therapies are being commercialized across major markets, including the U.S., EU, U.K., and Japan. Geographic expansion and label extensions are key drivers of revenue growth, indicating a broadening market presence for these treatments[2][5].

Conclusion

The market dynamics and financial trajectory for miglustat are characterized by steady growth, driven by strong patient demand, geographic expansion, and regulatory support. Key players like MYLAN LABORATORIES LTD, DIPHARMA SA, and NAVINTA LLC are crucial in this market, while companies like Amicus Therapeutics are driving the commercial success of miglustat through combination therapies.

Key Takeaways

• Market Growth: The global miglustat API market is projected to reach US$ 252.7 million by 2030, with a CAGR of 3.8% from 2024 to 2030.
• Key Players: MYLAN LABORATORIES LTD, DIPHARMA SA, and NAVINTA LLC are the main players in the miglustat API market.
• Applications: Miglustat is primarily used in capsule form and other formulations, targeting various rare diseases.
• Regulatory Status: Miglustat has been approved globally and designated as an orphan drug.
• Financial Performance: Amicus Therapeutics has shown robust revenue growth driven by strong demand for Galafold and Pombiliti + Opfolda.

FAQs

1. What is the projected market size for miglustat API by 2030?

   • The global market for miglustat API is forecasted to reach US$ 252.7 million by 2030[1].

2. Who are the main players in the miglustat API market?

   • The main players include MYLAN LABORATORIES LTD, DIPHARMA SA, and NAVINTA LLC[1].

3. What are the primary applications of miglustat?

   • Miglustat is used primarily in the treatment of glycogen storage disease type II, neurologic manifestations of Niemann-Pick diseases, Gaucher disease, and Niemann-Pick disease type C[4].

4. What is the mechanism of action of miglustat?

   • Miglustat targets the UGCG enzyme, reducing the accumulation of toxic substances in cells[4].

5. How has Amicus Therapeutics performed financially in relation to miglustat?

   • Amicus Therapeutics has reported strong revenue growth driven by Galafold and the successful launch of Pombiliti + Opfolda, which includes miglustat[2].

Sources

1. Miglustat API - Market Report: Valuates Reports.
2. Amicus Therapeutics Announces Full-Year 2023 Financial Results: Stock Titan.
3. Amicus Therapeutics Announces Full-Year 2022 Financial Results: Amicus Therapeutics Investor Relations.
4. Miglustat's R&D Progress and its Mechanism of Action on Drug Target: Synapse by Patsnap.
5. Amicus Therapeutics Announces Second Quarter 2023 Financial Results: Amicus Therapeutics Investor Relations.