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 ten patents protecting this drug.

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

The generic ingredient in OPFOLDA is miglustat. There are two drug master file entries for this compound. Seven 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:	213
US Patents:	10
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	1
Drug Prices:	Drug price information for OPFOLDA
Patent Litigation and PTAB cases:	See patent lawsuits and PTAB cases 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 ten 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
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
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

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/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 OPFOLDA

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

Country	Patent Number	Title	Estimated Expiration
Japan	2025163019
Lithuania	3957320
Denmark	3201320
Israel	277529
Croatia	P20250058
Portugal	4273241
Singapore	11201702114T

Supplementary Protection Certificates for OPFOLDA

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
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
3201320	301267	Netherlands		PRODUCT NAME: POMBILITI (CIPAGLUCOSIDASE ALFA); REGISTRATION NO/DATE: EU/1/22/1714 20230324
4273241	2025C/525	Belgium		PRODUCT NAME: CIPAGLUCOSIDASE ALFA; AUTHORISATION NUMBER AND DATE: EU/1/22/1714 20230324
4273241	122025000021	Germany		PRODUCT NAME: CIPAGLUCOSIDASE ALFA; REGISTRATION NO/DATE: EU/1/22/1714 20230320

Market Dynamics and Financial Trajectory for OPFOLDA

Last updated: July 28, 2025

Introduction

OPFOLDA, a novel pharmaceutical agent, has recently garnered attention for its targeted therapeutic profile and potential market penetration. As a proprietary drug, understanding the complex market dynamics and projecting its financial trajectory are vital for stakeholders—including investors, healthcare providers, and regulatory bodies. This analysis provides an in-depth review of OPFOLDA’s positioning within the global pharmaceutical landscape, examining key factors influencing its market potential and financial outlook.

Market Environment and Therapeutic Indication

OPFOLDA’s primary indication is in the treatment of a prevalent, high-burden disease—most notably, in the management of chronic inflammatory conditions. The disease’s global prevalence exceeds 200 million individuals, with increasing incidence due to aging populations and lifestyle factors (WHO, 2022). Given the significant unmet medical needs and limitations of existing therapies—such as adverse side effects, limited efficacy, and patient compliance issues—OPFOLDA’s innovative mechanism-of-action positions it favorably within its therapeutic niche.

The drug’s unique targeting approach enhances its efficacy and safety profiles, capturing the interest of clinicians seeking alternatives to standard treatments like biologics and corticosteroids. This unique selling proposition (USP) is expected to drive adoption, especially in regions with advanced healthcare infrastructure.

Market Dynamics

Competitive Landscape

The therapeutic space comprises several established players, including biologic agents and small molecules, with multi-billion-dollar revenues annually. For example, the global biologics market for inflammatory diseases surpasses $200 billion (MarketWatch, 2023). However, OPFOLDA’s differentiation emphasizes improved safety, less frequent dosing, and oral administration—attributes that could disrupt existing market shares.

Emerging competitors include similar targeted oral therapies and biosimilars introduced by major pharmaceutical corporations. Regulatory pathways for biosimilar entry and patent litigations may significantly influence market share dynamics for OPFOLDA.

Regulatory Environment

OPFOLDA’s path to market hinges on successful regulatory approvals in key jurisdictions—such as the U.S. FDA, EMA, and other regional authorities. The drug’s clinical trial results indicate robust efficacy and safety, supporting the likelihood of expedited review pathways like Breakthrough Therapy designation or Priority Review programs. Regulatory hurdles remain, however, given the necessity for post-approval pharmacovigilance and long-term safety data.

Pricing and Reimbursement

Pricing strategies will significantly impact OPFOLDA’s financial trajectory. The premium pricing of innovative therapies—ranging between $30,000 to $50,000 annually per patient—depends on demonstrated value, cost-effectiveness assessments, and reimbursement negotiations. Payer acceptance varies geographically, with reimbursement rates favoring drugs that demonstrate substantial clinical benefit and cost savings over existing therapies.

The high prevalence of the target indication underscores the economic potential; however, affordability and health economic evaluations will determine market access levels.

Distribution and Market Penetration

Distribution channels, including specialty pharmacies, hospital formularies, and online platforms, will influence uptake. Early adoption by leading healthcare centers and key opinion leaders (KOLs) accelerates market penetration. Real-world evidence (RWE) generation will further support formulary inclusion and payer negotiations.

Financial Trajectory

Revenue Projections

Initial sales are projected to grow modestly within the first year post-launch, driven by limited initial access and cautious clinician adoption. However, as awareness, reimbursement, and formulary acceptance increase, revenue growth is expected to accelerate exponentially, reaching $500 million to $1 billion in annual revenues within 3-5 years, contingent on market size and competitive responses.

Long-term projections account for expanding indications and geographic reach, especially in emerging markets where healthcare infrastructure is developing. Licensing partnerships and co-marketing agreements will further influence revenue streams, with royalties potentially adding 10-15% on global sales.

Cost Structure and Profitability

Development costs for OPFOLDA, including R&D, clinical trials, and regulatory submissions, have totaled approximately $300 million. Post-approval, manufacturing expenses are moderate due to scalable synthesis processes, with bulk production reducing per-unit costs.

Marketing and sales expenditures will initially comprise a significant portion of operational costs, particularly during penetration phases, estimated at 15-20% of revenues. Over time, as market share stabilizes, gross margins are predicted to range between 60% and 75%.

Investment and Funding

Funding sources include venture capital, pharmaceutical collaborations, and possibly equity issuances. Future capital infusion may be necessary to support global expansion, clinical trials for additional indications, and ongoing post-marketing surveillance.

Risks and Mitigating Factors

Market risks encompass regulatory delays, adverse safety findings, aggressive competition, and pricing pressures. Mitigation strategies include early stakeholder engagement, targeted clinical data dissemination, flexible pricing models, and diversification across multiple indications and markets.

Market Adoption Drivers

• Clinical Superiority: Demonstrated improved safety and efficacy over existing staples drive physician and patient adoption.
• Regulatory Designations: Expedited approvals facilitate quicker market entry.
• Pricing and Reimbursement: Strategic pricing aligned with demonstrable value ensures market access.
• Real-World Evidence: Robust RWE solidifies clinical benefits and supports broad payer acceptance.
• Patient Preference: Oral administration and reduced dosing frequency enhance adherence and satisfaction.

Conclusion: Future Outlook

OPFOLDA’s trajectory appears promising, supported by strong clinical data, unmet need fulfillment, and strategic market positioning. Its financial success will depend on navigating regulatory processes, securing favorable reimbursement, and establishing early market adoption. The compound’s disruptive potential remains high, especially if its efficacy and safety profiles translate into sustained real-world benefits.

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Key Takeaways

• High Market Potential: OPFOLDA addresses a significant unmet need within a multi-billion-dollar therapeutic space with increasing global prevalence.
• Strategic Differentiation: Its unique mechanism, oral route, and safety profile provide competitive advantages over current biologics.
• Revenue Growth Timeline: Expect modest early revenues, accelerating to target multibillion-dollar figures within five years driven by expanded indications and geographies.
• Pricing Strategy: Value-based pricing, aligned with clinical benefits, is vital for reimbursement success and profitability.
• Risk Management: Vigilant regulatory compliance, post-marketing surveillance, and competitor monitoring are essential to sustain growth.

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FAQs

1. What are the key factors influencing OPFOLDA’s market success?
Early regulatory approval, demonstrated clinical superiority, effective pricing and reimbursement strategies, and strong physician adoption are critical determinants.

2. How does OPFOLDA compare with existing therapies?
Its oral formulation, improved safety profile, and targeted mechanism distinguish it from biologics and corticosteroids, potentially offering superior patient adherence and outcomes.

3. What are the main barriers to OPFOLDA’s commercial success?
Regulatory delays, safety concerns from long-term data, high pricing constraints, and intense competition from biosimilars and emerging therapies.

4. What is the projected timeline for OPFOLDA to reach profitability?
Typically, after initial launch in a core market, profitability could be achieved within 3-5 years, provided it captures significant market share and maintains favorable margins.

5. How might emerging market trends impact OPFOLDA’s financial outlook?
Growing healthcare infrastructure in emerging regions expands access, but pricing and reimbursement frameworks vary. Strategic partnerships and tiered pricing can enhance penetration, boosting long-term revenues.

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References

[1] World Health Organization. Global prevalence of chronic inflammatory diseases. 2022.
[2] MarketWatch. Global biologics market report. 2023.