Ataluren

Drug Profile

Ataluren

Alternative Names: PTC 124; Translarna

Latest Information Update: 25 Mar 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
  • On Fast track

    Fast track status is assigned by the US FDA so therapies with the potential to address unmet needs can move faster through development.

    Cystic fibrosis; Duchenne muscular dystrophy
  • New Molecular Entity Yes

Highest Development Phases

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

Most Recent Events

  • 16 Mar 2017 PTC Therapeutics plans to discontinue development of Ataluren in Cystic fibrosis
  • 06 Mar 2017 Preregistration for Duchenne muscular dystrophy in USA (PO)
  • 06 Mar 2017 The US FDA sets PDUFA date of 24/10/2017 for NDA review for non-sense mutation Duchenne muscular dystrophy
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