What Amicus Therapeutics does

Amicus Therapeutics is a biopharmaceutical company that discovers, develops, and commercializes treatments for rare and genetic diseases, with a primary focus on lysosomal storage disorders. Founded in 2002 as a Mount Sinai spinout and headquartered in Princeton, NJ, the company has brought two therapies to market: Galafold (migalastat), an oral pharmacological chaperone approved in the U.S., EU, and Japan for Fabry disease in adults with amenable GLA variants; and Pombiliti (cipaglucosidase alfa-atga) administered with Opfolda (miglustat), a two-component enzyme replacement therapy approved in the U.S. and EU in 2023 for late-onset Pompe disease. The underlying technology platforms are pharmacological chaperone science for stabilizing deficient enzymes and recombinant enzyme replacement therapy combined with an oral enzyme stabilizer. Amicus also holds U.S. commercial rights to DMX-200, a Phase 3 investigational treatment for FSGS licensed from Dimerix, representing pipeline expansion into rare kidney disease.

The company generates revenue through specialty pharmaceutical distribution of its approved therapies, dispensed via specialty pharmacies and prescribed by specialists at treatment centers in over 70 countries, supported by 13 regional offices and the Amicus Assist patient support program. FY2025 revenue reached $634.2 million (17% constant-currency growth), with the company achieving non-GAAP net income of $96.8 million. Galafold holds U.S. patent exclusivity through January 2037, providing a long regulatory runway. As of April 2026, Amicus became a wholly owned subsidiary of BioMarin Pharmaceutical following a $4.8 billion all-cash acquisition, ending its independent public-company trajectory on NASDAQ (formerly FOLD). Prior to the acquisition, the company had approximately 251-500 employees and was treating more than 3,000 patients globally with its medicines.

Name

Amicus Therapeutics

Legal name

Amicus Therapeutics, Inc.

Website

https://amicusrx.com

Company type

Public

Founded year

2002

Operating status

Acquired

Headcount range

251–500 employees

Short description

Amicus Therapeutics is a biopharmaceutical company that develops and commercializes treatments for rare lysosomal storage disorders, primarily Galafold for Fabry disease and Pombiliti+Opfolda for Pompe disease, serving patients across 70+ countries. As of April 2026, Amicus operates as a wholly owned subsidiary of BioMarin Pharmaceutical following a $4.8 billion acquisition.

Ownership category

akta.pro rank

Product category

Rare Disease Biopharmaceuticals

NAICS

Pharmaceutical and Medicine Manufacturing (3254), Biological Product (except Diagnostic) Manufacturing (325414), Research and Development in Biotechnology (except Nanobiotechnology) (541714)

SIC

Pharmaceutical Preparations (2834), Biological Products, (No Disgnostic Substances) (2836)

akta.pro primary industry

Rare Pediatric & Congenital Disorder Therapies (HLAIAIAN)

Keywords

• Rare disease therapies
• Pharmacological chaperones
• Enzyme replacement therapy
• Lysosomal storage disorders
• Biopharmaceutical medicines

Headquarters

HQ city

Cranbury

HQ country

United States

HQ region

North America

Offices13 records

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel, Supply Chain, Operations, Marketing or Sales

Revenue model

1. Product Sales - Galafold: Galafold (migalastat) for Fabry disease generates revenue through prescription sales. Approved in U.S. (2018), EU (2016), and Japan (2018). Full-year 2025 revenues reported at $634.2 million representing 17% growth at constant exchange rates.
2. Product Sales - Pombiliti + Opfolda: Pombiliti (cipaglucosidase alfa-atga) + Opfolda (miglustat) for late-onset Pompe disease launched in U.S. and EU in 2023. Combined revenue contribution alongside Galafold drove 17% revenue growth in 2025.
3. Licensing Revenue - DMX-200: Amicus holds U.S. commercial rights to DMX-200, an investigational Phase 3 treatment for FSGS from Dimerix. This represents potential future revenue stream upon regulatory approval.

Go-to-market motion1 record

Distribution channels3 records

Marketing channels5 records

Core offering

Amicus Therapeutics discovers, develops, and commercializes medicines for rare and genetic diseases, with two FDA-approved therapies: Galafold (migalastat) for Fabry disease and Pombiliti + Opfolda for Pompe disease. The company also holds U.S. commercial rights to DMX-200, a Phase 3 candidate for FSGS licensed from Dimerix. Products are sold through specialty pharmacy channels and prescribed by specialists at rare disease treatment centers globally.

Product overview

Amicus Therapeutics is a biopharmaceutical company focused on developing and delivering transformative medicines for rare diseases. The company has brought forward two FDA-approved treatments: Galafold (migalastat), an oral pharmacological chaperone for Fabry disease, and Pombiliti (cipaglucosidase alfa-atga) used with Opfolda (miglustat), an enzyme therapy for Pompe disease. Additionally, the company holds U.S. rights to DMX-200, a Phase 3 investigational treatment for Focal Segmental Glomerulosclerosis (FSGS) licensed from Dimerix. In April 2026, Amicus was acquired by BioMarin Pharmaceutical Inc. for approximately $4.8 billion. The portfolio addresses lysosomal storage disorders affecting the kidneys, heart, muscles, and nervous system.

Differentiator

Problem solved

Functional benefit

Brands

• Amicus Assist: Patient support program providing personalized assistance with financial and insurance-related aspects of treatment
• Galafold
• Pombiliti
• Opfolda

Products and services

• Galafold (migalastat) Oral pharmacological chaperone therapy for adults with Fabry disease caused by amenable GLA gene variants. Binds to and stabilizes the deficient alpha-galactosidase A enzyme to reduce globotriaosylceramide (GL-3) accumulation in cells throughout the body, including the heart, kidneys, and nervous system. Approved in the U.S. (2018), EU (2016), and Japan (2018).
• Pombiliti (cipaglucosidase alfa-atga) + Opfolda (miglustat) Two-component enzyme replacement therapy for late-onset Pompe disease. Pombiliti (cipaglucosidase alfa-atga) is an intravenous recombinant enzyme replacement therapy, used with Opfolda (miglustat), an oral enzyme stabilizer that helps break down glycogen in lysosomes. Clinical study outcomes reported up to 90 months. Approved in the U.S. and EU in 2023.
• DMX-200 Investigational Phase 3 oral medicine for Focal Segmental Glomerulosclerosis (FSGS), a rare serious kidney disease causing progressive scarring in kidney filtering units and affecting over 40,000 people in the U.S. Amicus holds U.S. commercial rights licensed from Dimerix Limited and will be responsible for U.S. regulatory submissions. No FDA-approved specific therapies existed for FSGS as of November 2025.

Quantifiable outcome

• 17% revenue growth at constant exchange rates in FY2025
• +3 more outcomes

Named customers5 records

Segments4 records

Ideal customer profiles2 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature4 records

Partnerships

Two partnerships are on record, tiered minor and core.

• GenomenonminorStrategic or Co-development Partner · 10 March 2026Genomenon partnered with Amicus Therapeutics to improve Fabry disease diagnosis and research through curating genetic variant data. The collaboration resulted in submission of over 1,300 GLA gene variants to ClinVar, enhancing genetic test interpretation and reducing diagnostic delays.
• DimerixcoreStrategic or Co-development PartnerAmicus holds U.S. commercial rights to DMX-200, Dimerix's Phase 3 investigational treatment for Focal Segmental Glomerulosclerosis (FSGS). Amicus will be responsible for U.S. regulatory submissions. The drug targets FSGS, a rare kidney disease with over 40,000 U.S. patients and no cure.

Scale indicators7 records

Recent moves8 records

Expansion highlights5 records

Direct peers

• Ultragenyx Pharmaceutical: Commercial-stage rare disease biotech developing enzyme and gene therapies for genetic conditions. Highly comparable in business model: focused orphan indications, specialty distribution, and reliance on genetic identification to drive patient uptake.
• BridgeBio Pharma: Clinical and commercial-stage rare disease company developing therapies for genetic diseases. Comparable in pipeline focus on small-genetic-population therapies and rare nephrology/cardiomyology indications overlapping with Amicus's LSD focus.
• Sanofi (Genzyme): Markets Fabrazyme for Fabry disease and Nexviazyme/Myozyme for Pompe disease. Directly competes with Galafold and Pombiliti+Opfolda in the same lysosomal storage disorder indications with overlapping specialty prescriber channels.
• Swedish Orphan Biovitrum (Sobi): Dedicated rare disease biopharmaceutical company with franchises in genetic and metabolic disorders including enzyme replacement therapies. Comparable in target indications, distribution model, and European commercial footprint.
• Recordati Rare Diseases: Diversified rare disease business with metabolic and genetic disease therapies. Comparable in targeting orphan indications, specialty pharmacy distribution, and patient identification through disease awareness initiatives.
• Chiesi Farmaceutici (Chiesi Global Rare Diseases): Operates a global rare diseases division focused on lysosomal storage disorders and other genetic conditions. Comparable in LSD therapeutic focus, patient services infrastructure, and global commercial footprint across U.S. and Europe.

Broad incumbents

• Alexion Pharmaceuticals (AstraZeneca Rare Disease): Specializes in rare diseases including lysosomal and metabolic disorders. Comparable in rare disease commercial model, patient access infrastructure, and pipeline focus on genetic and enzyme-related conditions.
• Vertex Pharmaceuticals: Specialty biopharma with growing rare disease focus (cystic fibrosis franchise plus emerging genetic disease programs). Comparable business model of high-priced therapies for small genetic patient populations with deep payer engagement.
• Takeda Pharmaceutical: Through its Shire acquisition, markets Replagal for Fabry disease and has a broad rare disease franchise. Directly competes in Fabry and overlaps in rare disease commercial infrastructure and patient services programs.
• BioMarin Pharmaceutical: Acquirer of Amicus and a global rare disease biopharmaceutical company. Directly comparable as a rare disease-focused commercial-stage biotech with enzyme replacement therapies (e.g., Naglazyme, Vimizim, Aldurazyme) and a similar patient-identification model.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat4 records

Key risks6 records

Key highlights7 records

Customer concentration

• YouTubeyoutube.com/@BioMarinOfficial
• Facebookfacebook.com/357833324290284
• LinkedInlinkedin.com/company/amicus-therapeutics
• Instagraminstagram.com/biomarinpharmaceutical
• Xx.com/amicusrx1

Compliance3 records

Revenue estimate

Valuation estimate

Number of profiles

Profiles9 records

Subsidiaries11 records

Funding overview

Funding rounds20 records

Investors18 records

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A3 records

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about Amicus Therapeutics