Ataluren
- Generic Name
- Ataluren
- Brand Names
- Translarna
- Drug Type
- Small Molecule
- Chemical Formula
- C15H9FN2O3
- CAS Number
- 775304-57-9
- Unique Ingredient Identifier
- K16AME9I3V
- Background
Ataluren is a novel, orally administered drug that targets nonsense mutations. Ataluren is approved for use by the European Medicines Agency to treat Duchenne Muscular Dystrophy in patients aged 5 years and older who are able to walk. More specifically, ataluren is used in the small group of patients whose disease is caused by a specific genetic defect (called a ‘nonsense mutation’) in the dystrophin gene.
This drug does not yet have approval by the US Food and Drug Administration or by Health Canada for any indications.
- Indication
Ataluren is approved for use by the European Medicines Agency to treat Duchenne Muscular Dystrophy in patients aged 5 years and older who are able to walk. More specifically, ataluren is used in the small group of patients whose disease is caused by a specific genetic defect (called a ‘nonsense mutation’) in the dystrophin gene.
- Associated Conditions
- Duchenne Muscular Dystrophy (DMD)
- Associated Therapies
- -
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Earnings call: PTC Therapeutics raises 2024 revenue outlook
PTC Therapeutics updates 2024 revenue guidance to $750-$800 million following strong Q3 earnings, driven by DMD franchise. The company has over $1 billion in cash reserves and plans global launches for sepiapterin and vatiquinone. Challenges include Translarna's uncertain approval timelines in Europe and revenue variability from Translarna.
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FDA considers removing oral phenylephrine from OTC guidelines due to lack of efficacy, not safety concerns. Public comments invited until May 7, 2025, before final decision. Phenylephrine is in many nasal decongestants like Sudafed and Mucinex. FDA's proposal does not affect nasal spray decongestants.
FDA Accepts Resubmitted NDA for Ataluren in Nonsense Mutation Duchenne Muscular Dystrophy
FDA accepted PTC Therapeutics' resubmitted NDA for ataluren (Translarna) to treat nonsense mutation Duchenne muscular dystrophy (nmDMD), based on phase 3 trial data and STRIDE registry analyses. The therapy, approved in multiple countries, shows significant benefits in muscle function and safety. EU recently allowed ataluren to remain on market despite CHMP's negative opinion.
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PTC Therapeutics Announces FDA Acceptance of Resubmitted NDA for Translarna™ (ataluren) for Treating Nonsense Mutation Duchenne Muscular Dystrophy
PTC Therapeutics announced FDA's acceptance of the resubmitted NDA for Translarna™ (ataluren) for treating nonsense mutation Duchenne muscular dystrophy. The resubmission is based on significant benefits shown in Study 041 and the STRIDE registry, including delayed loss of ambulation and improved lung function. Translarna offers hope for a treatment targeting the underlying cause of nmDMD.
PTC Therapeutics Announces FDA Acceptance of Translarna NDA Resubmission
PTC Therapeutics announces FDA acceptance of Translarna NDA resubmission for nonsense mutation Duchenne muscular dystrophy (nmDMD). The NDA is supported by significant benefits in a global placebo-controlled trial and long-term treatment data from the STRIDE registry. Translarna aims to restore functional protein formation in patients with genetic disorders caused by nonsense mutations.
FDA Accepts Resubmission of Translarna NDA for Duchenne
FDA accepted Translarna's NDA resubmission for treating nmDMD, a fatal genetic disorder. Developed by PTC Therapeutics, Translarna aims to produce functional proteins in patients with nonsense mutations. Despite mixed trial results and regulatory challenges, it shows promise in delaying disease progression.
Drug Development Updates
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