ZAVESCA Drug Patent Profile

When do Zavesca patents expire, and when can generic versions of Zavesca launch?

Zavesca is a drug marketed by Actelion and is included in one NDA.

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

DrugPatentWatch^® Litigation and Generic Entry Outlook for Zavesca

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

Summary for ZAVESCA

US Patents:	0
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	2
Raw Ingredient (Bulk) Api Vendors:	64
Clinical Trials:	12
Patent Applications:	1,952
Drug Prices:	Drug price information for ZAVESCA
What excipients (inactive ingredients) are in ZAVESCA?	ZAVESCA excipients list
DailyMed Link:	ZAVESCA at DailyMed

See drug prices for ZAVESCA

Recent Clinical Trials for ZAVESCA

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

Sponsor	Phase
Actelion	Phase 4
Kashan University of Medical Sciences	Phase 3
Tehran University of Medical Sciences	Phase 3

See all ZAVESCA clinical trials

Pharmacology for ZAVESCA

Drug Class	Glucosylceramide Synthase Inhibitor
Mechanism of Action	Glucosylceramide Synthase Inhibitors

US Patents and Regulatory Information for ZAVESCA

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Exclusivity Expiration
Actelion	ZAVESCA	miglustat	CAPSULE;ORAL	021348-001	Jul 31, 2003	AB	RX	Yes	Yes

Expired US Patents for ZAVESCA

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 ZAVESCA

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 ZAVESCA

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

Country	Patent Number	Title	Estimated Expiration
Denmark	0698012
Denmark	1007043
World Intellectual Property Organization (WIPO)	9830219
Austria	268598
Australia	5813898
Spain	2097653

ZAVESCA Market Analysis and Financial Projection Experimental

Market Dynamics and Financial Trajectory for Zavesca

Introduction

Zavesca, also known as miglustat, is a drug primarily used to treat Gaucher’s disease and Niemann-Pick Type C (NPC) disease. Here, we will delve into the market dynamics and financial trajectory of Zavesca, highlighting its sales performance, market trends, and the impact of regulatory and clinical developments.

Approval and Clinical Use

Zavesca is approved by the FDA for the treatment of Gaucher’s disease Type 1, but it is also commonly used off-label for NPC, particularly in regions like the European Union, Canada, and Japan[1].

Sales Performance

Historical Sales

In 2016, Zavesca generated sales of 104 million Swiss francs, representing a 12% increase at constant exchange rates (CER) compared to 2015. This growth was driven by an increase in the number of patients treated for NPC, with a 13% rise in NPC patients receiving Zavesca globally[2].

Regional Sales

Sales of Zavesca varied by region:

• In the US, sales were strong, though impacted by the launch of generic miglustat for Gaucher’s disease Type 1.
• In Europe, sales were flat due to generic competition but maintained strong growth in the NPC indication.
• In Japan, Zavesca sales contributed to the overall growth, driven by the strong performance of other Actelion products as well[2].

Market Trends

Increasing Adoption for NPC

There has been a significant trend towards increased use of Zavesca in newly diagnosed NPC patients. Approximately 50% of US patients with NPC use Zavesca, with this figure rising to 70-80% for patients diagnosed within the past year. This trend is expected to continue as payer resistance to off-label use decreases[1].

Combination Therapy

Clinical evidence suggests that Zavesca works more effectively when used in combination with other treatments, such as arimoclomol, which is expected to be approved for NPC. This combination has shown a more significant reduction in disease severity, as indicated by the NPC composite clinical severity (NPC-CSS) score[1].

Financial Impact

Revenue Growth

The revenue from Zavesca has been a significant contributor to the financial performance of its manufacturers. For Actelion, the company that originally marketed Zavesca, the drug's sales were part of the overall strong financial results in 2016, with total product sales growing to CHF 2,412 million (+15% at CER)[2].

Market Size and Competition

The Gaucher disease drugs market, where Zavesca is a key player, was valued at USD 1.65 billion in 2022 and is expected to grow at a CAGR of 2.6% from 2023 to 2030. Zavesca, as a substrate replacement therapy (SRT), is one of the approved oral treatments for Gaucher’s disease, competing with other therapies like enzyme replacement therapy (ERT)[4].

Regulatory and Clinical Developments

FDA Approval and Off-Label Use

Although Zavesca is not FDA-approved for NPC, its off-label use for this condition is common and increasingly accepted by payers. The publication of clinical data, such as the Patterson and colleagues publication, has helped in securing insurance coverage for Zavesca in NPC patients[1].

Combination with Arimoclomol

The potential approval of arimoclomol for NPC is expected to further enhance the use of Zavesca. Clinical trials have shown that the combination of arimoclomol and Zavesca results in a more significant reduction in NPC-CSS scores compared to Zavesca alone, indicating a synergistic effect[1].

Key Players and Market Share

Zavesca is part of a broader market dominated by major pharmaceutical companies. Sanofi Genzyme, for instance, holds a significant market share in the Gaucher disease drugs market, but Zavesca's unique position as an SRT for both Gaucher’s and NPC diseases maintains its relevance and market presence[4].

Future Outlook

Continued Growth

Given the increasing acceptance and use of Zavesca for NPC, along with its established role in Gaucher’s disease, the drug is expected to continue contributing to the financial performance of its manufacturers. The growth in the number of patients diagnosed and treated for these rare diseases will likely sustain demand for Zavesca.

Competitive Landscape

The market for rare disease treatments is highly competitive, with ongoing research and development aimed at improving treatment options. However, Zavesca's established position and the potential for combination therapies will help it maintain its market share.

Conclusion

Zavesca's market dynamics are characterized by strong sales performance, increasing adoption for NPC, and a favorable financial trajectory. The drug's use in combination with other treatments, such as arimoclomol, enhances its clinical efficacy and market appeal. As the market for rare disease treatments continues to grow, Zavesca is poised to remain a significant player.

Key Takeaways

• Zavesca is approved for Gaucher’s disease Type 1 but is widely used off-label for NPC.
• Sales of Zavesca have shown steady growth, particularly in the NPC indication.
• The drug works more effectively in combination with other treatments like arimoclomol.
• Zavesca is part of a broader market for rare disease treatments, with a competitive landscape dominated by major pharmaceutical companies.
• The future outlook for Zavesca is positive, driven by increasing demand and acceptance for its use in NPC.

FAQs

Q: What is Zavesca primarily used for?

A: Zavesca, or miglustat, is primarily used to treat Gaucher’s disease Type 1 but is also commonly used off-label for Niemann-Pick Type C (NPC) disease.

Q: How has the sales performance of Zavesca been in recent years?

A: Zavesca has shown steady sales growth, particularly in the NPC indication, with a 12% increase at CER in 2016 compared to 2015.

Q: What is the impact of arimoclomol on Zavesca’s use?

A: Clinical trials suggest that arimoclomol works better in combination with Zavesca, leading to a more significant reduction in disease severity for NPC patients.

Q: Which regions have seen significant sales growth for Zavesca?

A: Sales growth has been notable in the US, Europe, and Japan, driven by increased use in NPC patients.

Q: What is the market outlook for Zavesca in the coming years?

A: The market outlook for Zavesca is positive, driven by increasing demand and acceptance for its use in NPC, as well as its established role in Gaucher’s disease.

Sources

1. Clinical Trials Arena: Orphazyme's arimoclomol to be used with Zavesca after FDA approval[1].
2. GlobeNewswire: Actelion announces excellent financial results for 2016[2].
3. Amicus Therapeutics: Amicus Therapeutics Reports Preliminary 2022 Revenue and Provides 2023 Strategic Outlook[3].
4. Grand View Research: Gaucher Disease Drugs Market Size & Share Report, 2030[4].