Ataluren is an orally delivered, investigational new drug discovered by PTC Therapeutics. The drug is being developed as a new approach for the treatment of nonsense mutation genetic disorders. Nonsense mutations create a premature stop signal in the mRNA causing the ribosome to terminate translation before a full-length protein is generated. This causes the protein to be truncated and non-functioning. Ataluren is designed to allow the ribosome to continue translation of the mRNA, overriding the premature stop signal and leading to the formation of a functioning protein. Ataluren is a potential therapy for patients with nonsense mutation genetic disorders.
Ataluren has demonstrated proof of concept in Phase 2a clinical trials and is currently in a registration-directed pivotal study with the goal of demonstrating that allowing expression of a critical missing protein will safely provide clinical benefits for patients with nonsense mutation genetic disorders. Ataluren has been generally well tolerated across all clinical studies to date.
The FDA and the European Commission have granted PTC124 (ataluren) orphan drug status for the treatment of DMD and cystic fibrosis due to nonsense mutations. The FDA has also granted PTC124 (ataluren) Subpart E designation for expedited development, evaluation, and marketing.
PTC has an exclusive collaboration with Genzyme Corporation for the development and commercialization of ataluren. PTC Therapeutics will market ataluren in the United States and Canada, while Genzyme will commercialize the product in other regions of the world. The development of ataluren has also been supported by grants from the Cystic Fibrosis Foundation Therapeutics Inc. (the nonprofit affiliate of the Cystic Fibrosis Foundation), FDA's Office of Orphan Products Development, the Muscular Dystrophy Association, the Parent Project Muscular Dystrophy, and the
|SOURCE PTC Therapeutics, Inc.|
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