A biopharmaceutical company focused on the discovery and development of small-molecule drugs targeting post-transcriptional control mechanisms--PTC Therapeutics, Inc. (PTC)-- has been awarded a two-year grant from the Office of Orphan Products Development (OOPD) Grant Program of the USFDA.
The said grant will support the patient-related costs of the company's ongoing U.S.-based Phase 2 clinical trial of PTC124 for the treatment of nonsense-mutation mediated Duchenne muscular dystrophy (DMD).
According to Stuart Peltz, Ph.D., President and CEO of PTC Therapeutics:
"It is an honor to receive this grant from the FDA in support of our clinical development efforts. 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 2 CF program. Similarly, this new award will support the development of PTC124 as a potential treatment for DMD."
DMD patients lack dystrophin, a protein that is critical to the structural stability of muscle fibers. PTC124 is currently under a Phase 2 DMD clinical trial initiated by PTC in January 2006.
Preliminary data from the said study will be presented at the Parent Project UK Muscular Dystrophy 4th International DMD conference today (Saturday, October 21st).
Read more at the PTC press release.
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