migalastat
migalastat: developed by 1 company · 6 clinical trials tracked · 1 FDA decision.
migalastat evidence translation
What we can confirm
1 FDA record · 6 tracked trials
What it means
What to watch
Prepared research question
How could migalastat → FOLD affect its developer, and what evidence supports the link?
Traceable citations · Unknowns marked
See linked entities and source evidence3 indications · 2 study sponsors
Related indications
Development Pipeline (2)
| Company | Phase |
|---|---|
| AMICUS THERAPEUTICS, INC. (FOLD) Fabry disease (treatment of adults with a confirmed diagnosi… | Approved |
| AMICUS THERAPEUTICS, INC. (FOLD) Fabry disease in patients with amenable GLA variants | Approved |
Clinical Trials (6)
A Prospective, Observational Study of Patients With Fabry Disease (US Specific)
Primary endpoint: Annualized rate of change in Estimated Glomerular Filtration Rate (eGFR)
An 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
Primary endpoint: Safety: Incidence of TEAEs, SAEs, and AEs leading to discontinuation of study drug
A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding
Primary endpoint: Number of major birth defects
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
Primary endpoint: Maximum observed concentration (Cmax)
A Randomized, Open-label, Parallel-group, 18-month Phase 3 Study to Evaluate the Effect of Venglustat Compared With Usual Standard of Care on Left Ventricular Mass Index in Participants With Fabry Disease and Left Ventricular Hypertrophy
Primary endpoint: Slope of left ventricular mass index as measured by cardiac magnetic resonance imaging (MRI) (central reading)
A Long-term, Open-label Study to Evaluate the Safety, Pharmacodynamics, and Efficacy of Migalastat in Subjects > 12 Years of Age With Fabry Disease and Amenable GLA Variants
Primary endpoint: Incidence of Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and AEs (Adverse Events) Leading to Discontinuation of Study Drug
FDA Decisions1 records
Frequently asked questions
Who is developing migalastat?
migalastat is being developed by AMICUS THERAPEUTICS, INC..
How many clinical trials involve migalastat?
BioSniper tracks 6 clinical trials involving migalastat.
What is migalastat's latest FDA decision?
The most recent tracked FDA decision for migalastat is Approval for 1 INDICATIONS AND USAGE GALAFOLD is indicated for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene ( GLA ) variant based on in vitro assay data [see Dosage and Administration (2.1) and Clinical Pharmacology (12.1) ] . This indication is approved under accelerated approval based on reduction in kidney interstitial capillary cell globotriaosylceramide (KIC GL-3) substrate [see Clinical Studies (14) ] . Continued approval for this indicati, dated August 10, 2018.