MIGALASTAT HYDROCHLORIDE - Generic Drug Details

What are the generic drug sources for migalastat hydrochloride and what is the scope of freedom to operate?

Migalastat hydrochloride is the generic ingredient in one branded drug marketed by Amicus Therap Us and is included in one NDA. There are sixty-three patents protecting this compound. Additional information is available in the individual branded drug profile pages.

Migalastat hydrochloride has two hundred and eighty-five patent family members in thirty-one countries.

One supplier is listed for this compound.

Summary for MIGALASTAT HYDROCHLORIDE

International Patents:	285
US Patents:	63
Tradenames:	1
Applicants:	1
NDAs:	1
Finished Product Suppliers / Packagers:	1
Raw Ingredient (Bulk) Api Vendors:	41
Clinical Trials:	21
Patent Litigation and PTAB cases:	See patent lawsuits and PTAB cases for MIGALASTAT HYDROCHLORIDE
What excipients (inactive ingredients) are in MIGALASTAT HYDROCHLORIDE?	MIGALASTAT HYDROCHLORIDE excipients list
DailyMed Link:	MIGALASTAT HYDROCHLORIDE at DailyMed

Recent Clinical Trials for MIGALASTAT HYDROCHLORIDE

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

Sponsor	Phase
Amicus Therapeutics	PHASE3
Genzyme, a Sanofi Company	Phase 3
Amicus Therapeutics	Phase 1

See all MIGALASTAT HYDROCHLORIDE clinical trials

Anatomical Therapeutic Chemical (ATC) Classes for MIGALASTAT HYDROCHLORIDE

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

Paragraph IV (Patent) Challenges for MIGALASTAT HYDROCHLORIDE

Tradename	Dosage	Ingredient	Strength	NDA	ANDAs Submitted	Submissiondate
GALAFOLD	Capsules	migalastat hydrochloride	123 mg	208623	3	2022-08-10

US Patents and Regulatory Information for MIGALASTAT HYDROCHLORIDE

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	GALAFOLD	migalastat hydrochloride	CAPSULE;ORAL	208623-001	Aug 10, 2018		RX	Yes	Yes	11,633,387			Y
Amicus Therap Us	GALAFOLD	migalastat hydrochloride	CAPSULE;ORAL	208623-001	Aug 10, 2018		RX	Yes	Yes	11,622,962			Y
Amicus Therap Us	GALAFOLD	migalastat hydrochloride	CAPSULE;ORAL	208623-001	Aug 10, 2018		RX	Yes	Yes	8,592,362				Y
Amicus Therap Us	GALAFOLD	migalastat hydrochloride	CAPSULE;ORAL	208623-001	Aug 10, 2018		RX	Yes	Yes	11,357,764			Y
Amicus Therap Us	GALAFOLD	migalastat hydrochloride	CAPSULE;ORAL	208623-001	Aug 10, 2018		RX	Yes	Yes	10,925,866
Amicus Therap Us	GALAFOLD	migalastat hydrochloride	CAPSULE;ORAL	208623-001	Aug 10, 2018		RX	Yes	Yes	11,357,763
Amicus Therap Us	GALAFOLD	migalastat hydrochloride	CAPSULE;ORAL	208623-001	Aug 10, 2018		RX	Yes	Yes	10,406,143

International Patents for MIGALASTAT HYDROCHLORIDE

Country	Patent Number	Title	Estimated Expiration
Mexico	2009011473	REGIMENES DE DOSIFICACION PARA EL TRATAMIENTO DE ENFERMEDADES POR ALMACENAMIENTO LISOSOMAL QUE UTILIZAN CHAPERONAS FARMACOLOGICAS. (DOSING REGIMENS FOR THE TREATMENT OF LYSOSOMAL STORAGE DISEASES USING PHARMACOLOGICAL CHAPERONES.)
European Patent Office	4062915	MIGALASTAT ATTACHÉ À ALPHA GALACTOSIDASE A (MIGALASTAT BOUND TO ALPHA GALACTOSIDASE A)
Taiwan	I775453
South Korea	20220116305
Japan	6837469
Taiwan	202118490
Japan	2025143262

Supplementary Protection Certificates for MIGALASTAT HYDROCHLORIDE

Patent Number	Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
2787345	53/2016	Austria		PRODUCT NAME: MIGALASTAT ODER EIN SALZ DAVON, EINSCHLIESSLICH DES HYDROCHLORIDSALZES; REGISTRATION NO/DATE: EU/1/15/1082 (MITTEILUNG) 20160531
2787345	C02787345/01	Switzerland		PRODUCT NAME: MIGALASTAT; REGISTRATION NO/DATE: SWISSMEDIC-ZULASSUNG 66108 28.10.2016
2787345	2016/050	Ireland		PRODUCT NAME: MIGALASTAT OR A SALT THEREOF, INCLUDING THE HYDROCHLORIDE SALT; REGISTRATION NO/DATE: EU/1/15/1082 20160526
2787345	1690052-4	Sweden		PRODUCT NAME: MIGALASTAT OR A SALT THEREOF, INCLUDING THE HYDROCHLORIDE SALT.; REG. NO/DATE: EU/1/15/1082 20160531
2787345	300843	Netherlands		PRODUCT NAME: MIGALASTAT OF EEN ZOUT DAARVAN, WAARONDER HET HYDROCHLORIDEZOUT; REGISTRATION NO/DATE: EU/1/15/1082 20160531
2787345	SPC/GB16/067	United Kingdom		PRODUCT NAME: MIGALASTAT OR A SALT THEREOF, INCLUDING THE HYDROCHLORIDE SALT.; REGISTERED: UK EU/1/15/1082/001 20160531
2787345	132016000116282	Italy		PRODUCT NAME: MIGALASTAT O UN SUO SALE, COMPRESO IL SALE CLORIDRATO(GALAFOLD); AUTHORISATION NUMBER(S) AND DATE(S): EU/1/15/1082, 20160531

Market Dynamics and Financial Trajectory for Migalastat Hydrochloride

Last updated: July 27, 2025

Introduction

Migalastat hydrochloride, marketed primarily under the brand name Galafold, is an oral pharmacological chaperone approved by the U.S. Food and Drug Administration (FDA) in 2018 for the treatment of Fabry disease, a rare X-linked lysosomal storage disorder caused by mutations in the GLA gene. Since its launch, migalastat's market dynamics and financial trajectory have been shaped by factors including clinical efficacy, regulatory landscape, competitive forces, and evolving payer policies. This comprehensive analysis explores these dimensions, providing insights critical for stakeholders ranging from pharmaceutical developers to investors.

Market Overview

Manufacturing and Regulatory Status

Migalastat is developed by Amicus Therapeutics, a biotechnology firm with a focused portfolio on rare diseases. Its mechanism involves stabilizing certain mutant forms of alpha-galactosidase A, facilitating enzyme activity in Fabry patients with amenable mutations. Regulatory clearance in major markets, including the EU and Japan, complements FDA approvals, broadening market access.

Patient Population and Market Potential

Fabry disease affects approximately 1 in 40,000 to 117,000 males worldwide, with variability depending on diagnosed cases. The disease's rarity classifies migalastat within the Orphan Drug domain, enabling market exclusivity protections and certain incentives. Yet, the proportion of patients with mutations responsive to migalastat remains a critical determinant of market penetration — approximately 35-50% of Fabry patients possess amenable mutations [1].

Competitive Landscape

Previous standard treatments include enzyme replacement therapies (ERT) such as agalsidase alfa and beta, which require biweekly infusions. Migalastat offers the advantage of oral administration; however, its adoption hinges on whether it matches ERT in efficacy, safety, and patient compliance.

Market Dynamics

Clinical and Adoption Trends

Real-world data indicates that migalastat has gained traction among patients with amenable mutations seeking less invasive treatment options. The shift toward oral therapy has favored its adoption, particularly among younger patients and those with logistical barriers to infusions. Nonetheless, clinicians often reserve migalastat for specific patient subsets due to its mutation-specific efficacy profile.

Regulatory and Reimbursement Environment

Approval timelines and reimbursement policies significantly influence market penetration. Payers' acceptance depends on evidence demonstrating cost-effectiveness and comparative efficacy. Studies suggest that in healthcare systems where oral treatments are prioritized for their convenience, migalastat benefits from favorable coverage, boosting sales.

Pricing Strategies and Market Penetration

Pricing of migalastat remains competitive relative to ERTs, with an average annual cost around $370,000 in the US, comparable to ERTs. Health authorities and insurance providers scrutinize this expense against the clinical benefits, impacting uptake rates.

Market Challenges

Key challenges include limited mutation responsiveness, which restricts eligible patient populations, and competition from emerging therapies, including gene therapy candidates, which may disrupt long-term treatment paradigms.

Financial Trajectory

Revenue Generation and Growth

Since its 2018 approval, migalastat has demonstrated steady, albeit modest, revenue growth. In 2021, Amicus reported global net product sales of approximately $127 million, with the U.S. contributing around 60% [2]. The growth trajectory reflects adoption rates, mutation eligibility, and market expansion efforts.

Forecasting Future Revenue

Projections indicate a compound annual growth rate (CAGR) of 10-15% over the next five years, contingent on broader mutation screening programs, expansion into new geographic markets, and long-term clinical data reinforcing its position as a first-line therapy for amenable mutation patients.

Impacts of Pipeline and Market Changes

The potential introduction of modified or new therapies could pressure migalastat's market share. Conversely, expanding mutation diagnostic methods could increase eligible patient pools, positively influencing demand.

Key Market Drivers

• Patient Preference for Oral Therapy: Growing demand for non-infusion treatments fuels consideration and adoption.
• Regulatory Approvals in Emerging Markets: Greater access in Asia-Pacific and Latin America broadens income streams.
• Mutation Screening Initiatives: Enhancing diagnostic capabilities increases identified treatable patients.
• Long-term Clinical Data: Positive safety and efficacy outcomes reinforce market confidence.

Barriers to Growth

• Limited Mutational Spectrum: Only patients with amenable mutations benefit, capping market size.
• High Treatment Costs: Expense concerns may hinder widespread adoption in resource-constrained settings.
• Competition from ERTs and Emerging Modalities: ERTs remain entrenched, and gene therapies threaten future landscape.

Conclusion

Migalastat hydrochloride's market dynamics are characterized by cautiously optimistic growth, driven by its convenience as an oral therapy and targeted mechanism. Its financial trajectory depends on mutation screening expansion, reimbursement policies, and competitive developments. While the current market is somewhat constrained by mutation-specific eligibility, ongoing clinical data and regulatory expansions could significantly enhance its commercial outlook in the coming years.

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Key Takeaways

• Selective Market Penetration: Migalastat’s success hinges on increasing mutation screening to identify eligible patients—not all Fabry patients qualify.
• Growing Adoption for Quality-of-Life Advantages: As patients and clinicians prioritize non-invasive treatments, migalastat's role is set to expand within its niche.
• Pricing and Reimbursement: Competitive pricing aligned with payer strategies is critical to sustain growth, especially in emerging markets.
• Pipeline and Competition: Future innovations, including gene therapy, could challenge migalastat’s market share, emphasizing the importance of ongoing clinical validation.
• Regulatory Expansion Opportunities: Broader approvals and labeling updates are potential catalysts for revenue acceleration.

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FAQs

1. What determines whether a Fabry patient is eligible for migalastat?
Eligibility depends on the presence of specific amenable GLA mutations confirmed through genetic testing. Approximately 35-50% of Fabry patients have these mutations, limiting the scope of treatment.

2. How does migalastat compare with enzyme replacement therapies?
Migalastat offers an oral, daily therapy alternative, avoiding infusion-related complications. However, its efficacy is mutation-dependent, and in some cases, ERT may still be preferred.

3. What is the global market potential for migalastat?
Major growth hinges on expanding mutation testing infrastructure, regulatory approvals in new regions, and reimbursement strategies. Currently, the U.S. accounts for the majority of revenues.

4. How could emerging therapies impact migalastat’s financial outlook?
Gene therapies and novel modalities could overcome mutation-specific limitations or offer curative prospects, potentially diminishing migalastat’s market share over time.

5. What factors influence the pricing of migalastat?
Efficacy, treatment convenience, competitor pricing, and payer negotiations directly impact its cost to healthcare systems, affecting adoption levels.

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Sources:

1. Desnick RJ, et al. (2018). "Migalastat: a pharmacological chaperone for Fabry disease." Molecular Genetics and Metabolism [1].
2. Amicus Therapeutics Annual Report (2021). Financial and clinical data.