Rare Diseases

Rare Pediatric Disease and Fast Track Designations Granted to Gene Therapy for Myotubular Myopathy

Audentes Therapeutics, Inc. has been granted both Rare Pediatric Disease and Fast Track Designation by the U.S. Food and Drug Administration (FDA) for its gene therapy product candidate AT132.

The drug, intended to treat X-Linked Myotubular Myopathy (XLMTM), had previously received Orphan Drug Designation from both the FDA and the European Medicines Agency (EMA).

XLMTM is usually evident at birth, as patients with it typically have decreased muscle tone and muscle weakness. The condition affects the muscles vital for movement, and is diagnosed almost exclusively in males. The XLMTM patient population commonly suffers from breathing issues, resulting in most patients living only into early childhood.

“The incentives provided by the Rare Pediatric Disease and Fast Track designations are significant and include opportunities to work with FDA to expedite the development of AT132, and the potential to obtain a valuable Rare Pediatric Disease Priority Review Voucher upon approval,” Matthew R. Patterson, President and Chief Executive Officer of Audentes said via press release. “We are eager to leverage these benefits and to work closely with the FDA and XLMTM patient community as we pursue our goal of developing AT132 to treat this devastating…