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Ataluren - PTC Therapeutics

Drug Profile

Ataluren - PTC Therapeutics

Alternative Names: PTC 124; Translarna

Latest Information Update: 17 Oct 2019

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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
  • New Molecular Entity Yes

Highest Development Phases

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

Most Recent Events

  • 07 Oct 2019 PTC Therapeutics withdraws a phase II trial in Aniridia (PO) as the trial was cancelled before protocol was implemented ( NCT04117880)
  • 06 Aug 2019 PTC Therapeutics intends to request a re-examination with CHMP for an opinion in October 2019
  • 26 Jul 2019 PTC Therapeutics requests a re-examination of the CHMP negative opinion on the approval of ataluren for Duchenne muscular dystrophy in European Union
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