biologics license application

Amicus Begins Rolling FDA Submission to Advance AT-GAA Therapy for LOPD

Amicus Therapeutics is seeking U.S. approval of AT-GAA, its investigational therapy for late-onset Pompe disease. The company has begun a rolling submission of a biologics license application (BLA) to the U.S. Food and Drug Administration (FDA). This allows Amicus to submit individual sections of the application as soon they are…

Amicus Completes Patient Enrollment for Phase 3 Trial of AT-GAA; Remains on Track to File BLA in 2021

Amicus Therapeutics has completed and exceeded patient enrollment for its Phase 3 PROPEL trial of AT-GAA, its investigational chaperone therapy for the treatment of Pompe disease. The company also remains on track to file a biologics license application…