Ataluren - PTC Therapeutics

Drug Profile

Ataluren - PTC Therapeutics

Alternative Names: PTC 124; Translarna

Latest Information Update: 08 Nov 2017

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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; Methylmalonic acidaemia

Most Recent Events

  • 02 Nov 2017 PTC Therapeutics files a dispute resolution request with the US FDA regarding the Complete Response Letter for ataluren in Duchenne muscular dystrophy
  • 25 Oct 2017 PTC Therapeutics receives complete response letter from the FDA for ataluren in Duchenne muscular dystrophy
  • 06 Jul 2017 PTC Therapeutics initiates enrolment in the phase II Study 041 trial for Duchenne muscular dystrophy (In children, In adolescents, In adults, In the elderly) in USA (NCT03179631)
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