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Top-line Data From Phase 3 Trial of Ataluren in Patients with Nonsense Mutation Cystic Fibrosis Show Promising Results
Date:6/8/2012

V1). The secondary objective was to assess pulmonary exacerbation rates for ataluren compared to placebo. Additional endpoints evaluated other aspects of patient function, drug activity, and safety. The 48-week trial enrolled 238 patients, ages six years and older, at multiple sites in North America, Europe, and Israel. Patients were randomly assigned to one of two treatment arms: ataluren (10 mg/kg morning, 10 mg/kg midday, 20 mg/kg evening) or placebo (morning, midday, evening). Patients who completed the study were eligible to receive open-label ataluren in an ongoing extension study. 

About AtalurenAn investigational new drug, ataluren is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation. A nonsense mutation is an alteration in the genetic code that prematurely halts the synthesis of an essential protein. The resulting disorder is determined by which protein cannot be expressed in its entirety and is no longer functional, such as the cystic fibrosis transmembrane conductance regulator protein (CFTR) in nmCF.

The FDA and the European Commission have granted ataluren Orphan Drug status for the treatment of nonsense mutation cystic fibrosis and nonsense mutation Duchenne and Becker muscular dystrophy. The FDA has also granted ataluren Subpart E designation for expedited development, evaluation, and marketing for CF and dystrophinopathy and Fast Track designation for the development of treatment for nonsense mutation dystrophinopathy.

The development of ataluren has been supported by grants from Cystic Fibrosis Foundation Therapeutics Inc. (the nonprofit affiliate of the Cystic Fibrosis Foundation); Muscular Dystrophy Association; FDA's Office of Orphan Products Development; National Center for Research Resources; National Heart, Lung, and Blood Institute; and Parent Project Muscular Dystrophy.

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