Drug Profile


Alternative Names: PTC 124; Translarna

Latest Information Update: 12 Jan 2017

Price : $50

At a glance

  • Originator PTC Therapeutics
  • Developer PTC Therapeutics; Sanofi Genzyme
  • Class 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
  • Preregistration Cystic fibrosis
  • Phase II Aniridia; Dravet syndrome; Epilepsy; Mucopolysaccharidosis I
  • Suspended Haemophilia A; Haemophilia B; Methylmalonic acidaemia

Most Recent Events

  • 09 Jan 2017 PTC Therapeutics intends to file the NDA for ataluren for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) over protest with the US FDA in the first quarter of 2017
  • 09 Jan 2017 European Commission ratifies positive CHMP opinion for the annual renewal of conditional marketing authorisation of Ataluren for Duchenne muscular dystrophy (In adults, In children) in European Union
  • 11 Nov 2016 The CHMP recommends renewal of marketing authorisation for Duchenne muscular dystrophy
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