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 seven patents protecting this drug.

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

The generic ingredient in OPFOLDA 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 Opfolda

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

Summary for OPFOLDA

International Patents:	137
US Patents:	7
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	1
Drug Prices:	Drug price information 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 seven 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	11,753,632
Amicus Therap Us	OPFOLDA	miglustat	CAPSULE;ORAL	215211-001	Sep 28, 2023		RX	Yes	Yes	10,208,299		Y	Y
Amicus Therap Us	OPFOLDA	miglustat	CAPSULE;ORAL	215211-001	Sep 28, 2023		RX	Yes	Yes	10,961,522

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/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

International Patents for OPFOLDA

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

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

Supplementary Protection Certificates for OPFOLDA

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

OPFOLDA Market Analysis and Financial Projection Experimental

Market Dynamics and Financial Trajectory for Pombiliti + Opfolda

Introduction to Pombiliti + Opfolda

Pombiliti + Opfolda, a treatment for adults living with Pompe disease, has been a significant focus for Amicus Therapeutics since its approval in the US, EU, and the UK in mid-2023. This combination therapy consists of cipaglucosidase alfa (Pombiliti) and miglustat (Opfolda), offering a new treatment option for a rare and devastating genetic disorder.

Regulatory Approvals and Launch

The commercial launch of Pombiliti + Opfolda has been successfully underway in the three largest markets. The therapy received approvals from regulatory bodies such as the FDA, MHRA, and EU authorities in 2023, marking a crucial milestone in its market entry[3][4].

Patient Uptake and Demand

As of the end of 2023, approximately 120 patients were on treatment with commercial product or scheduled to be treated. This strong patient demand indicates a positive reception of the therapy among both patients and healthcare providers. The patient uptake has continued to grow, with a significant number of patients switching from other treatments like Nexviazym and Lumizyme[1][4].

Revenue Performance

2023 Revenue

In 2023, Pombiliti + Opfolda generated net product sales of $11.6 million, with $8.5 million of that coming in the fourth quarter. This initial revenue was a promising start for the newly launched therapy[1][4].

2024 Revenue Projections

For 2024, Amicus Therapeutics has set a revenue target for Pombiliti + Opfolda between $62 million and $67 million. However, due to strong performance, this guidance has been increased to $69 million to $71 million. The second quarter of 2024 saw revenue of $15.9 million, a 44% increase from the first quarter of 2024[2][5].

Quarterly Growth

The third quarter of 2024 saw further growth, with Pombiliti + Opfolda revenue rising to $21 million, representing a 33% increase compared to the second quarter of 2024. This robust growth underscores the therapy's increasing market acceptance and demand[5].

Market Projections

Global Pompe Disease Market

The global Pompe disease market is projected to grow significantly, from approximately $1.3 billion in 2023 to around $2 billion by 2030. Pombiliti + Opfolda is poised to be a major player in this growing market, especially given its approval and strong launch performance[3].

Market Sales Split

The market for Pompe disease treatments is split across different regions, with the US accounting for 43%, the EU for 36%, and other regions for 21%. This geographic distribution highlights the potential for Pombiliti + Opfolda to capture a substantial share of the global market[3].

Competitive Landscape

Pombiliti + Opfolda competes with other enzyme replacement therapies (ERTs) such as Nexviazym and Lumizyme. However, its unique combination of cipaglucosidase alfa and miglustat offers a distinct therapeutic approach, which has attracted a significant number of patients switching from other treatments. As of the third quarter of 2024, about 68% of patients in the US were switching from Nexviazym, indicating a strong competitive position for Pombiliti + Opfolda[5].

Financial Impact on Amicus Therapeutics

Total Revenue Growth

The strong performance of Pombiliti + Opfolda has contributed to Amicus Therapeutics' overall revenue growth. The company's total revenue for the second quarter of 2024 was $126.7 million, a 34% year-over-year increase. This growth has led to an increase in the company's full-year revenue growth guidance to 26% to 31% on a constant currency basis[2].

Cash Reserves and Operating Expenses

As of December 31, 2023, Amicus Therapeutics had cash reserves of $286.2 million. Despite significant investments in research and development, the company is managing its expenses effectively, with non-GAAP operating expenses guidance reduced due to efficient commercial execution[2][5].

Future Outlook

Non-GAAP Profitability

Amicus Therapeutics aims to achieve non-GAAP profitability in the first full year of the launch of Pombiliti + Opfolda. With strong revenue growth and managed operating expenses, the company is on track to meet this goal. For 2025, the company anticipates working through previously expensed inventory, which may affect gross margins but is expected to stabilize in the mid-80s for the full year[5].

Regulatory and Market Expansion

The company continues to make progress on expanding commercial access to Pombiliti + Opfolda through multiple regulatory submissions. As reimbursement and launches in additional countries are secured, the therapy is expected to further penetrate the global market[5].

Key Takeaways

• Strong Launch Performance: Pombiliti + Opfolda has seen robust revenue growth since its launch, with significant patient uptake.
• Revenue Projections: The therapy is projected to generate between $69 million and $71 million in revenue for 2024.
• Market Growth: The global Pompe disease market is expected to grow to $2 billion by 2030, providing a substantial growth opportunity for Pombiliti + Opfolda.
• Competitive Position: The therapy is attracting patients switching from other treatments, indicating a strong competitive position.
• Financial Impact: The strong performance of Pombiliti + Opfolda is contributing to Amicus Therapeutics' overall revenue growth and financial stability.

FAQs

Q: What is Pombiliti + Opfolda used for?

A: Pombiliti + Opfolda is a treatment for adults living with Pompe disease, a rare genetic disorder.

Q: When was Pombiliti + Opfolda approved?

A: Pombiliti + Opfolda was approved in the US, EU, and the UK in mid-2023.

Q: What are the revenue projections for Pombiliti + Opfolda in 2024?

A: The revenue target for Pombiliti + Opfolda in 2024 is between $69 million and $71 million.

Q: How many patients are currently on Pombiliti + Opfolda treatment?

A: As of the end of 2023, approximately 120 patients were on treatment with commercial product or scheduled to be treated.

Q: What is the expected growth of the global Pompe disease market?

A: The global Pompe disease market is expected to grow from approximately $1.3 billion in 2023 to around $2 billion by 2030.

Sources

1. Amicus Therapeutics Reports Preliminary 2023 Revenue and ... - IR.AmicusRx.com
2. Amicus Therapeutics Announces Second Quarter 2024 Financial ... - StockTitan.net
3. By: Owen Brinker and Adam Rose - CMICH.edu
4. Amicus Therapeutics Announces Full-Year 2023 Financial Results ... - IR.AmicusRx.com
5. Earnings call: Amicus Therapeutics reports robust Q3 growth, raises ... - Investing.com