Amicus Therapeutics

NYSE/NASDAQ:FOLDBiotech

Philadelphia, Pennsylvania, United States · Est. 2002

Amicus Therapeutics is a biopharmaceutical company focused on developing therapies for rare genetic diseases, particularly lysosomal storage disorders.

Pipeline Programs↗

Distinct drugs in development (Preclinical–Phase 3)

Indications↗

Distinct diseases across all programs

Clinical Trials↗

All sponsored trials (all statuses)

Orange Book Patents

Across 1 drug with upcoming expirations

Pipeline

Pipeline Snapshot

By Phase

5 drugs in development

Phase 34

Phase 21

Preclinical1

By Therapeutic Area

top 1

Rare Disease

583%

R&D Momentum

40/100

Pipeline Velocity

11/100

Trial Completion Rate

85/100

Pipeline Concentration

72/100

Pipeline Freshness

75/100

Activity

Recent Activity

Last 14 days

Jul 11, 2026

Recruiting

A Prospective, Observational Study of Patients With Fabry Disease (US Specific)

Jul 7, 2026

Active, not recruiting

An Open-label Study to Evaluate the Safety and Pharmacokinetics of Migalastat HCl in Subjects With Fabry Disease and Amenable GLA Variants and Severe Renal Impairment or End-Stage Renal Disease Treated With Hemodialysis

Earlier

Jun 26, 2026TrialAn Open-label Study of the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of 12 Month Treatment With Migalastat in Pediatric Subjects (Aged 2 to < 12 Years) With Fabry Disease and Amenable GLA Variants

Feb 20, 2026EX-99.1AMICUS THERAPEUTICS, INC. (FOLD) (CIK 0001178879)

Mar 18, 2025TrialA Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding

Feb 16, 2024TrialA Global Prospective Observational Registry of Patients With Pompe Disease

Jul 18, 2023TrialA Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18

Mar 1, 2020TrialA Study to Describe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease With Enzyme Replacement Therapy.

4 more events

Feb 13, 2020TrialZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD

Dec 18, 2019TrialA Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LOPD)

Oct 11, 2019TrialSafety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease

Dec 4, 2018TrialA Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease