PTC Therapeutics Completes Enrollment of Pivotal Trial in Muscular...( - Ataluren designated as generic name...),Health

- Ataluren designated as generic name for PTC124 -

SOUTH PLAINFIELD, N.J., Feb. 3 /PRNewswire/ -- PTC Therapeutics, Inc. (PTC) today announced that it has successfully completed patient enrollment of its pivotal clinical trial of PTC124 in patients with nonsense mutation Duchenne and Becker muscular dystrophy (nmDMD/BMD). Accrual was completed two months ahead of schedule. The company also announced today that PTC124 has been issued the generic name, ataluren.

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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 nmDMD/BMD. It will also evaluate ataluren's long-term safety profile over a longer term than in previous studies. The pivotal trial is ongoing at 37 sites on four continents and in 11 countries. The study has enrolled 174 patients across the United States, Europe, Canada, Australia, and Israel.

"This trial represents a critical milestone for the entire DMD/BMD community - 20 years after sequencing the dystrophin gene we have the first pivotal study for a potential disease-modifying therapy," said Brenda Wong, MD, associate professor of pediatrics and neurology, Cincinnati Children's Hospital Medical Center. "The rapid accrual is a testament to the commitment of boys with DMD/BMD and their families and the strong sense of urgency on the part of so many investigational teams around the world."

"Duchenne/Becker muscular dystrophy is a rare disorder and patients whose disease is due to a nonsense mutation represent only 13 percent of these cases, which created an enrollment challenge. However, the collaborative approach taken by clinical trial sites, advocacy groups, and PTC Therapeutics
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