Ataluren by PTC Therapeutics for Duchenne Muscular Dystrophy: Likelihood of Approval
Ataluren is under clinical development by PTC Therapeutics and currently in Phase III for Duchenne Muscular Dystrophy. According to GlobalData, Phase III drugs for Duchenne Muscular Dystrophy have a 62% phase transition success rate (PTSR) indication benchmark for progressing into Pre-Registration. GlobalData's report assesses how Ataluren's drug-specific PTSR and Likelihood of Approval (LoA) scores compare to the indication benchmarks. Buy the report here.GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data. Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval.Ataluren overviewAtaluren (Translarna) is a benzoic acid derivative acts against muscular dystrophy. It is formulated as granules and powder for suspension for oral route of administration. Translarna is indicated for the treatment of duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene, in ambulatory patients aged 5 years and older and also in non ambulatory patients. Ataluren is under development for the treatment of nonsense mutation becker muscular dystrophy, aniridia, shwachman-diamond syndrome (SDS) and Dravet syndrome. It was also under development for nonsense mutation cystic fibrosis (nmCF), mucopolysaccharidosis I (Hurler syndrome), spinal muscular atrophy, Rett syndrome and nonsense mutation hemophilia A & B (nmHA/B).PTC Therapeutics overviewPTC Therapeutics discovers, develops, and markets medicines for the treatment of rare disorders. Its marketed products include Evrysdi (risdiplam) for spinal muscular atrophy, Translarna (ataluren) and Emflaza (deflazacort) for the treatment of Duchenne muscular dystrophy. The company's pipeline candidates target the treatment of Huntington’s disease; children with inherited mitochondrial disease; leiomyosarcoma; acute myelogenous leukemia; and aromatic L-amino acid decarboxylase deficiency. PTC Therapeutics works in collaboration with various biotechnology and pharmaceutical companies for advancing its pipeline candidates. It operates through its subsidiaries in the US and Europe. PTC Therapeutics is headquartered in South Plainfield, New Jersey, the US. For a complete picture of Ataluren's drug-specific PTSR and LoA scores, buy the report here.
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