Ataluren

Drug Profile

Ataluren

Alternative Names: PTC 124; Translarna

Latest Information Update: 27 Jul 2017

$50 / €47 *
* $ price applies to USA, Canada and Latin America. € price applies to the rest of the world. Final gross prices may vary according to local VAT.

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

  • 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)
  • 06 Jun 2017 The Advisory Committee Meeting for non-sense mutation Duchenne muscular dystrophy is tentatively scheduled for September 28, 2017
  • 29 May 2017 PTC Therapeutics plans the Study 041 for Duchenne muscular dystrophy (In children, In adolescents, In adults, In the elderly) in European Union in June 2017 (NCT03179631)
Restricted Access

Oops, it looks like you don’t have a valid subscription to this content. To gain full access to the content and functionality of the AdisInsight database try one of the following.

  • with a username/password associated to an account with a valid subscription
  • Contact your organization’s admin about adding this content to your AdisInsight subscription
  • Request a trial

If you are a subscriber to this content then contact us at AsktheExpert.AdisInsight@springer.com so we can help.

Back to top