PTC124 is an orally delivered, investigational new drug discovered by PTC Therapeutics. The drug is being developed for the treatment of genetic disorders due to nonsense mutations. Nonsense mutations are single-point alterations in the genetic code that prematurely stop the translation process, leading to production of truncated, non-functional proteins. PTC124 induces the cellular translation machinery to read through nonsense mutations, inducing production of full-length, functional proteins. PTC124 has demonstrated proof of concept in phase 2a clinical trials. Across all clinical studies to date, PTC124 has been generally well tolerated. PTC124 is currently in phase 2b development with the goal of demonstrating that increasing functional protein levels in patients with nonsense-mediated genetic disorders will safely provide clinical benefits.
PTC124 has been granted orphan drug status by the FDA and the European Commission for the treatment of CF and DMD due to nonsense mutations. The FDA has also granted PTC124 Subpart E designation for expedited development, evaluation, and marketing.
PTC has an exclusive collaboration with Genzyme Corporation to develop and commercialize PTC124 outside the U.S. and Canada. The development of PTC124 has also been supported by grants from, the Muscular Dystrophy Association, Parent Project Muscular Dystrophy, FDA's Office of Orphan Products Development, the National Center for Research Resources and notably, the Cystic Fibrosis Foundation Therapeutics Inc. (the nonprofit affiliate of the Cystic Fibrosis Foundation), which recently expanded support of PTC124 to include funding up to $25 million.
|SOURCE PTC Therapeutics, Inc.|
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