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news.Biopharmaceutical company PTC Therapeutics has been granted fast track designation from the FDA for the development of a drug designed to treat Duchenne muscular dystrophy due to a nonsense mutation in the dystrophin gene.
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The drug candidate, called PTC124, was granted orphan drug designation for the treatment of Duchenne muscular dystrophy (DMD) in December 2004.
PTC124 is currently in phase II clinical trials in DMD and cystic fibrosis (CF) in cases in which a nonsense mutation is the cause of the disease. PTC expects to complete these phase II clinical trials in the second half of 2006.
The fast track program is designed to facilitate the development and expedite the review of new drugs that are intended to treat serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs. Development programs receiving fast track designations typically receive FDA priority review, shortening the review period from a typical ten months to six.
“Fast track designation in DMD is an additional important element in the development of PTC124,” said Dr Stuart Peltz, president and CEO of PTC. “DMD is an unmet medical need where only palliative options are currently available. We hope PTC124 will represent a therapeutic option for patients with DMD due to a nonsense mutation.”
PTC124 is an orally delivered investigational product candidate in development for the treatment of genetic disorders due to nonsense mutations. Nonsense mutations are single-point alterations in the genetic code that prematurely halt the translation process, producing a shortened, non-functional protein.