OOPD grant from FDA for PTC Therapeutics

New Jersey, USA-based PTC Therapeutics, a biopharmaceutical company focused on the discovery and development of small-molecule drugs targeting post-transcriptional control mechanisms, says it has been awarded a two-year grant from the Office of Orphan Products Development (OOPD) Grant Program of the Food and Drug Administration. The grant will support the patient-related costs of the company's ongoing USA-based Phase II clinical trial of PTC124 for the treatment of nonsense-mutation-mediated Duchenne muscular dystrophy.

Stuart Peltz, the firm's chief executive, said: "last year, PTC was awarded a grant from the FDA for the development of PTC124 as a potential treatment for cystic fibrosis (CF), which we have used to advance our Phase II CF program. Similarly, this new award will support the development of PTC124 as a potential treatment for DMD."

PTC initiated the Phase II DMD clinical trial in January. Patients with DMD lack dystrophin, a protein that is critical to the structural stability of muscle fibers. This multi-site, open-label, dose-ranging clinical trial is evaluating muscle dystrophin expression in patients with nonsense-mutation-mediated DMD. Blood levels of muscle-derived creatine kinase are being measured as assessments of muscle integrity. PTC124 safety, compliance, and pharmacokinetics are also being evaluated, the company said.