Ataluren

Drug Profile

Ataluren

Alternative Names: PTC 124; Translarna

Latest Information Update: 31 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
  • 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

  • 24 Mar 2017 Discontinued - Phase-III for Cystic fibrosis (In adolescents, In children, In adults) in Israel, Canada, Australia, Argentina, Brazil, USA (PO)
  • 24 Mar 2017 Regulatory submission withdrawn for Cystic fibrosis (In children, In adults) in European Union due to negative results from the ACT CF (PO)
  • 16 Mar 2017 PTC Therapeutics plans to discontinue development of Ataluren in Cystic fibrosis
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