PTC Therapeutics Initiates Registration-Directed Phase 3 Trial of Ataluren in Cysti...(uren (PTC124(R)) Ataluren is the fir...)

Ataluren is the first investigational new drug designed to restore the formation of a functioning protein in patients with genetic disorders due to a nonsense mutation. A nonsense mutation is an alteration in the genetic code that prematurely halts the synthesis of an essential protein. Ataluren is currently being investigated for use in patients with nmCF and nmDMD/BMD.

Ataluren has been granted orphan drug status for the treatment of nmCF and nmDMD/BMD by the U.S. Food and Drug Administration (FDA) and the European Commission. The FDA has also granted ataluren Subpart E designation for expedited development, evaluation, and marketing. The development of ataluren has been supported by the Cystic Fibrosis Foundation Therapeutics Inc. (the nonprofit affiliate of the Cystic Fibrosis Foundation), the FDA Office of Orphan Products Development, the Muscular Dystrophy Association, Parent Project Muscular Dystrophy, and the National Center for Research Resources.

Completed Ataluren Clinical Trials

Data from Phase 2a clinical trials of ataluren in pediatric and adult patients with nmCF show that administration of ataluren results in production of functional CFTR and statistically significant improvements in CFTR chloride channel function in the airways. Ataluren treatment is also associated with reductions in cough frequency and improvements in pulmonary function tests.

Across all ataluren clinical trials to date, including Phase 1 healthy-volunteer trials, ataluren has been generally well tolerated. In Phase 2a trials in nmCF and nmDMD/BMD, adverse events have been largely consistent with background symptoms and have usually been mild. No concerning adverse findings have been identified based on physical examinations, vital sign measurements, electrocardiograms, or laboratory studies. The mean compliance with ataluren therapy has been greater than 90 percent in all
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