Amicus Therapeutics Advances Pediatric Fabry Disease Study with Migalastat

Amicus Therapeutics ((FOLD)) announced an update on their ongoing clinical study.

Amicus Therapeutics is conducting a clinical study titled 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. The study aims to evaluate the safety and efficacy of migalastat in young patients with Fabry disease, a rare genetic disorder. This research is significant as it targets a vulnerable pediatric population, potentially offering a new treatment avenue.

The intervention being tested is Migalastat HCl 20 mg, administered as dispersible tablets every other day. The drug is designed to treat Fabry disease by stabilizing the body’s own dysfunctional enzyme, offering an alternative to enzyme replacement therapy.

This Phase 3b study is open-label and uncontrolled, involving a single group of participants. It is structured in two treatment stages over 12 months, followed by an open-label extension. The primary purpose is treatment, with no masking involved.

The study is currently recruiting, with an estimated start date in July 2025. The primary completion date is yet to be determined, and the last update was submitted in July 2025. These dates are crucial for tracking the study’s progress and potential market entry.

The update on this study could influence Amicus Therapeutics’ stock performance positively, as successful results may enhance investor confidence and market position. The study’s focus on a pediatric population could differentiate Amicus from competitors, potentially impacting the broader industry landscape.

The study is ongoing, with further details available on the ClinicalTrials portal.