Audentes : FDA Lifts Clinical Hold On Trial Of AT132 For Treatment Of X-Linked Myotubular Myopathy

(RTTNews) - Audentes Therapeutics, an Astellas genetic medicines company, said that the U.S. Food and Drug Administration lifted the clinical hold for the ASPIRO clinical trial evaluating AT132 in patients with X-linked myotubular myopathy.

X-linked myotubular myopathy or XLMTM is a serious, life-threatening neuromuscular disease characterized by extreme muscle weakness, respiratory failure, and early death.

The company said it is working to complete all clinical and regulatory activities necessary to resume dosing and plans to have discussions at a future date with the regulators on the path forward toward global registration filings for AT132.

AT132 has been granted Regenerative Medicine and Advanced Therapy, Rare Pediatric Disease, Fast Track, and Orphan Drug designations by the U.S. Food and Drug Administration, and Priority Medicines and Orphan Drug designations by the European Medicines Agency.