Ataluren - PTC Therapeutics

Drug Profile

Ataluren - PTC Therapeutics

Alternative Names: PTC 124; Translarna

Latest Information Update: 16 Jul 2018

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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; Gene modulators; 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

  • 10 Jul 2018 Positive efficacy and adverse events data from a phase II trial in Duchenne muscular dystrophy released by PTC Therapeutics
  • 01 Jun 2018 The Committee for Medicinal Products for Human Use (CHMP) recommends expansion of the ataluren (Translarna™ ) label to include patients as young as 2 years of age with nonsense mutation Duchenne muscular dystrophy (nmDMD)
  • 20 Feb 2018 The Office of New Drugs of the US FDA denies PTC Therapeutics' appeal of the Complete Response Letter in relation to ataluren's NDA for nonsense mutation Duchenne muscular dystrophy
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