PTC THERAPEUTICS ANNOUNCES EUROPEAN MEDICINES AGENCY VALIDATION OF MARKETING AUTHORIZATION APPLICATION FOR ATALUREN IN DUCHENNE MUSCULAR DYSTROPHY
Enrollment for a Global Confirmatory Phase 3 Clinical Trial Planned for 1Q13
SOUTH PLAINFIELD, NJ – December 6, 2012 – PTC Therapeutics, Inc. (PTC) today announced that the European Medicines Agency (EMA) has validated a Marketing Authorization Application (MAA) seeking conditional approval for ataluren, an investigational new drug for the treatment of patients with nonsense mutation Duchenne muscular dystrophy (nmDMD). Validation of the MAA confirms that the submission is complete and begins the EMA’s Committee for Human Medicinal Products’ (CHMP) review process. Ataluren is the only treatment currently in clinical development targeting the cause of disease in patients with a nonsense mutation.
“Ataluren is a promising potential therapy for nonsense mutation Duchenne muscular dystrophy,” stated Dr. Thomas Voit, Medical and Scientific Director, Institut de Myologie. “PTC has developed a standard for DMD clinical trials and now the DMD community can share in the achievement of the first MAA ever filed for DMD. We appreciate PTC’s commitment to the clinical development of ataluren for this severe disorder for which only palliative treatment options currently exist.”