Amicus Therapeutics
53
5
8
33
Key Highlights
Risk & Performance
Pipeline Risk Assessment
Pipeline Risk Assessment
Based on historical performance
Moderate Risk
Score: 45/100
15.1%
8 terminated/withdrawn out of 53 trials
80.5%
-6.0% vs industry average
26%
14 trials in Phase 3/4
76%
25 of 33 completed trials have results
Key Signals
Enrollment Performance
Analytics
Activity Timeline
Global Presence
Clinical Trials (53)
A Study of Patients With Fabry Disease (US Specific)
Role: lead
A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants
Role: lead
A Global Prospective Observational Registry of Patients With Pompe Disease
Role: lead
A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LOPD)
Role: lead
Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease
Role: lead
A Study to Describe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease With Enzyme Replacement Therapy.
Role: lead
A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18
Role: lead
A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease
Role: lead
ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD
Role: lead
First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221
Role: lead
Expanded Access for ATB200/AT2221 for the Treatment of Pompe Disease
Role: lead
Study to Evaluate the Safety of AT2220 (Duvoglustat) in Pompe Disease
Role: lead
A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease
Role: lead
Expanded Access for ATB200/AT2221 for the Treatment of IOPD
Role: lead
A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding
Role: lead
STRIDE Study - A Study in Subjects With LOPD Who Are Currently Being Treated With ERT
Role: lead
Physician Initiated Expanded Access Request for Migalastat in Individual Patients With Fabry Disease
Role: lead
Drug-drug Interaction Study
Role: lead
Understanding Fabry Disease Therapy Choices Through the Eyes of the Patients
Role: lead
Real World Evidence Study of Danish Fabry Patients
Role: collaborator