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news.Can ataluren improve walking, muscle function in muscular dystrophy patients?
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PTC Therapeutics, a biopharmaceutical company, has successfully completed patient enrollment in its pivotal clinical trial of PTC124 in patients with nonsense mutation Duchenne and Becker muscular dystrophy.
Accrual was completed two months ahead of schedule. The company has also announced that PTC124 has been issued the generic name, ataluren.
The pivotal trial is going on at 37 sites on four continents and in 11 countries. The study has enrolled 174 patients across the US, Europe, Canada, Australia, and Israel.
The one-year, multi-center, randomized, double-blind, placebo-controlled study is designed to determine whether ataluren can improve walking, muscle function and strength in patients with nonsense mutation Duchenne and Becker muscular dystrophy (nmDMD/BMD). It will also evaluate ataluren’s long-term safety profile over a longer term than in previous studies.
According to the company, this is the first pivotal trial for ataluren. In addition, PTC is conducting an open-label, long-term safety study in boys with nmDMD who participated in an early Phase IIa trial. PTC also plans to initiate a second pivotal clinical trial of ataluren in patients with nonsense mutation cystic fibrosis in 2009.
Stuart Peltz, president and CEO of PTC Therapeutics, said: We are pleased to have completed enrollment of our first pivotal study of ataluren in boys and young men with nmDMD/BMD. We look forward to completing this study over the next year as we continue to work to develop additional innovative therapies for the muscular dystrophy community.