Amicus Therapeutics
Amicus Therapeutics works on 6 rare diseases tracked on Trial Friend, including Epidermolysis Bullosa, Fabry Disease, Familial Hypertrophic Cardiomyopathy and 3 more, with 7 active clinical trials (5 recruiting) and 4 FDA-approved rare disease drugs.
Amicus Therapeutics is a Philadelphia-based biotech that spent two decades building a focused portfolio in lysosomal storage disorders, a family of rare genetic diseases where missing or defective enzymes allow toxic substances to accumulate inside cells. Founded in 2002, Amicus traded publicly on the NASDAQ under the ticker FOLD until April 27, 2026, when BioMarin Pharmaceutical completed its acquisition of the company for $14.50 per share in an all-cash deal valued at $4.8 billion. Amicus is now a wholly owned subsidiary of BioMarin.
The flagship product Amicus brought to the deal is Galafold (migalastat), which became the first oral treatment for Fabry disease when it was approved in 2018. Fabry disease is a rare X-linked genetic disorder where the body cannot properly break down a fatty substance called globotriaosylceramide, which then accumulates and damages the heart, kidneys, and nervous system. Before Galafold, the only available therapies were lifelong intravenous enzyme replacement infusions. Galafold is taken as a pill every other day and works only in patients with specific amenable GLA variants, which a genetic test must confirm before treatment. The drug generated $458 million in 2024 sales, and under a 2024 patent settlement Teva may sell a generic version in the U.S. starting January 30, 2037.
The other major asset is Pombiliti + Opfolda, a two-component therapy for late-onset Pompe disease. Pompe is a rare lysosomal storage disorder where the body cannot break down glycogen properly, which leads to progressive muscle weakness and respiratory decline. Pombiliti (cipaglucosidase alfa) is a recombinant enzyme replacement, and Opfolda (miglustat) is an oral stabilizer that helps the enzyme reach muscle tissue more effectively. The combination is approved for adults with late-onset Pompe disease who weigh at least 40 kg and are not improving on their current enzyme replacement therapy.
In addition to the marketed drugs, BioMarin gained U.S. rights to DMX-200 through the acquisition. DMX-200 is an investigational small molecule in Phase 3 development for focal segmental glomerulosclerosis (FSGS), a rare and progressive kidney disease. Under BioMarin, the legacy Amicus pipeline now sits alongside one of the largest rare disease infrastructures in the industry, with expanded global reach and manufacturing capacity for Galafold, Pombiliti, and Opfolda for the patients who need them.
Amicus Therapeutics Drug Pipeline
Amicus Therapeutics has 7 active clinical trials across 2 development stages, with 5 currently recruiting participants. Clinical trials advance through phases: Phase 1 tests safety in a small group, Phase 2 evaluates effectiveness and side effects, Phase 3 confirms benefit in a larger population, and Phase 4 monitors long-term safety after FDA approval.
Note: This pipeline counts the active and recruiting trials listed on this page, from ClinicalTrials.gov. For companies that work outside rare disease, we list only trials for rare diseases we track, so the company's full pipeline may be larger.
Amicus Therapeutics Clinical Trials (7)
Active and recruiting clinical trials sponsored by Amicus Therapeutics, sourced live from ClinicalTrials.gov. Each trial card shows the study phase, current recruitment status, conditions under investigation, study locations, eligibility criteria, and a direct link to the full ClinicalTrials.gov record. You can also download a one-page PDF summary to share with your doctor.
Note: Recruitment statuses on ClinicalTrials.gov may not immediately reflect recent FDA decisions, sponsor announcements, or enrollment changes. Always confirm a trial's current status directly with the study coordinator.
Amicus Therapeutics FDA-Approved Drugs (4)
Medications developed or marketed by Amicus Therapeutics that have received U.S. Food and Drug Administration approval. Drug data is sourced from the openFDA database and includes brand names, generic names, approval dates, and matched rare disease indications. Where a drug treats a condition covered by Trial Friend, the disease name links directly to that disease page.
| Drug Name | Brand Name | Rare Diseases | Approval Date |
|---|---|---|---|
| MIGALASTAT HYDROCHLORIDE | Galafold oral | Aug 10, 2018 | |
| MIGLUSTAT Glucosylceramide Synthase Inhibitor [EPC] | OPFOLDA oral | Sep 28, 2023 | |
| CIPAGLUCOSIDASE ALFA-ATGA | POMBILITI ATGA intravenous | Sep 28, 2023 | |
| CIPAGLUCOSIDASE ALFA + MIGLUSTAT | POMBILITI + OPFOLDA | — |
Amicus Therapeutics Trial Locations
Amicus Therapeutics clinical trials are running at 105 sites in 18 countries. Click any country to drill down by state, city, and individual research facility. Proximity to a trial site is one of the most important factors in deciding whether to participate.
Rare Disease Focus Areas (6)
Diseases targeted by Amicus Therapeutics's clinical trial and drug development programs
Epidermolysis bullosa is a group of rare genetic blistering disorders caused by mutations affecting proteins anchoring the epidermis to the dermis. Fragile skin blisters and erodes with minimal trauma...
Fabry disease is a rare inherited lysosomal storage disorder where a missing enzyme causes fatty substances called globotriaosylceramide to accumulate in cells throughout the body. This buildup damage...
Familial hypertrophic cardiomyopathy is a genetic heart muscle disease characterized by inappropriate left ventricular hypertrophy and diastolic dysfunction. Caused by mutations in genes encoding sarc...
Gaucher disease is a rare inherited lysosomal storage disorder caused by deficiency of the enzyme glucocerebrosidase, resulting in accumulation of fatty substances in the spleen, liver, and bone marro...
Glycogen Storage Disease Type II (GSD II, also called Pompe Disease) is a lysosomal storage disorder caused by deficiency of the enzyme acid alpha-glucosidase (GAA), which breaks down glycogen. This l...
Pompe disease is a rare inherited lysosomal storage disorder caused by deficiency of the enzyme acid alpha-glucosidase, leading to excessive accumulation of glycogen in muscles and organs. This buildu...
Patient Resources
Organizations and resources related to Amicus Therapeutics's rare disease focus areas
Frequently Asked Questions About Amicus Therapeutics
Common questions about Amicus Therapeutics's rare disease programs, clinical trials, and treatments.