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Patients Can't Clear Translarna's Data Hurdles As PTC Falls Short At FDA Panel

 
• By Sue Sutter

Patient testimony to the contrary, advisory committee members were not persuaded that PTC Therapeutics' ataluren, which failed its primary endpoint in two trials, is effective in treating nonsense mutation Duchenne muscular dystrophy.

MUSCULAR DYSTROPHY words written on label tag with medicine,syringe,keyboard and stethoscope

Compelling personal testimony about the clinical benefits experienced by Duchenne muscular dystrophy (DMD) patients taking PTC Therapeutics Inc.'s Translarna (ataluren) was not enough to overcome less-than-compelling randomized clinical trial data, a US FDA advisory committee concluded Sept. 28.

Ten of 11 members of the Peripheral and Central Nervous System Drugs Advisory Committee voted that while it is possible ataluren may be effective in treating dystrophinopathies, particularly DMD, resulting from nonsense mutations in the dystrophin

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