Ataluren - PTC Therapeutics

Drug Profile

Ataluren - PTC Therapeutics

Alternative Names: PTC 124; Translarna

Latest Information Update: 28 Feb 2018

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

Most Recent Events

  • 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
  • 28 Jan 2018 No recent reports of development identified for phase-I development in Mucopolysaccharidosis-I in USA (PO, Suspension)
  • 17 Jan 2018 Discontinued - Phase-II for Methylmalonic acidaemia in United Kingdom, Italy, Belgium, Switzerland, France, Germany (PO)
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