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Ataluren - PTC Therapeutics

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Drug Profile

Ataluren - PTC Therapeutics

Alternative Names: PTC 124; Translarna

Latest Information Update: 11 Jan 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 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

  • 27 Aug 2018 PTC Therapeutics plans a phase II trial for Duchenne muscular dystrophy in November 2018 (NCT03648827)
  • 10 Jul 2018 Positive efficacy and adverse events data from a phase II trial in Duchenne muscular dystrophy released by PTC Therapeutics
  • 24 Jun 2018 Biomarkers information updated
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