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Ataluren - PTC Therapeutics

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Drug Profile

Ataluren - PTC Therapeutics

Alternative Names: PTC 124; Translarna

Latest Information Update: 08 Nov 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; Muscular dystrophies; Spinal muscular atrophy
  • 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

  • 30 Oct 2024 The US FDA accepts for review the resubmitted New Drug Application (NDA) for Translarna™ (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD)
  • 30 Oct 2024 Efficacy data from the phase III Study 041 trial in Duchenne muscular dystrophy released by PTC Therapeutics
  • 18 Oct 2024 Committee for Medicinal Products for Human Use (CHMP) maintains a negative opinion following the re-examination procedure for the conditional marketing authorization of Ataluren (Translarna™)

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