Advertise With UsAdvertise on our extensive network of industry websites and newsletters.
Get the PBR newsletterSign up to our free email to get all the latest PBR
news.Biopharmaceutical firm PTC Therapeutics has been awarded a two-year grant from the Office of Orphan Products Development Grant Program of the FDA for its product for Duchenne muscular dystrophy, PTC124.
Subscribe to our email newsletter
The grant will support the patient-related costs of the company’s ongoing phase II clinical trial of PTC124. PTC initiated the phase II clinical trial of PTC124 in January 2006.
Patients with Duchenne muscular dystrophy (DMD) lack dystrophin, a protein that is critical to the structural stability of muscle fibers. This phase II trial is evaluating muscle dystrophin expression in patients with nonsense-mutation-mediated DMD. PTC124 safety, compliance, and pharmacokinetics are also being evaluated.
Last year PTC was awarded a grant from the FDA for the development of cystic fibrosis in support of a phase II study.
PTC’s current pipeline of clinical and preclinical product candidates addresses multiple indications, including genetic disorders, oncology, and infectious diseases.