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Ataluren - PTC Therapeutics

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Drug Profile

Ataluren - PTC Therapeutics

Alternative Names: PTC 124; Translarna

Latest Information Update: 14 Aug 2024

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At a glance

  • Originator PTC Therapeutics
  • Developer PTC Therapeutics; Sanofi Genzyme
  • Class Antianaemics; Antiepileptic drugs; Antifibrotics; Antihaemorrhagics; Eye disorder therapies; Oxadiazoles; Small molecules
  • Mechanism of Action Cystic fibrosis transmembrane conductance regulator stimulants; Protein synthesis stimulants
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Cystic fibrosis; Aniridia; Duchenne muscular dystrophy; Mucopolysaccharidosis I; Spinal muscular atrophy; Muscular dystrophies
  • New Molecular Entity Yes

Highest Development Phases

  • Marketed Duchenne muscular dystrophy
  • Phase II Mucopolysaccharidosis I
  • Preregistration Submission Withdrawal Cystic fibrosis
  • Suspended Haemophilia A; Haemophilia B
  • Discontinued Aniridia; Dravet syndrome; Epilepsy; Methylmalonic acidaemia

Most Recent Events

  • 08 Aug 2024 PTC resubmits the NDA for ataluren in Duchenne muscular dystrophy
  • 28 Jun 2024 Committee for Medicinal Products for Human Use (CHMP) issues a negative opinion following the re-examination procedure for the conditional marketing authorization of Ataluren (Translarna™)
  • 20 May 2024 European Commission (EC) decides not to adopt the CHMP's negative opinion on the annual renewal for the conditional marketing authorization of Ataluren

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