Translarna

Expired

This medicine's authorisation has expired

ataluren

Overview

On 28 March 2025, the European Commission issued a decision to not renew the conditional marketing authorisation for Translarna (ataluren) in the European Union (EU). The marketing authorisation holder for the medicine was PTC Therapeutics International Limited.

The non-renewal of the marketing authorisation follows the opinion issued by EMA’s human medicines committee, CHMP, which recommended to not renew the authorisation because the effectiveness of the medicine has not been confirmed.

Further information is available in the news announcement published at the time of the CHMP opinion.

Translarna was granted marketing authorisation in the EU on 31 July 2014 for the treatment of Duchenne muscular dystrophy.

The European Public Assessment Report (EPAR) for Translarna will be updated to indicate that the marketing authorisation is no longer valid.

Translarna : EPAR - Medicine overview

Translarna : EPAR - Risk-management-plan summary

Translarna : EPAR - Risk-management-plan summary

Product information

Translarna : EPAR - Product Information

Translarna : EPAR - All Authorised presentations

Product details

• Name of medicine:
  Translarna
• Active substance:
  ataluren
• International non-proprietary name (INN) or common name:
  ataluren
• Therapeutic area (MeSH):
  Muscular Dystrophy, Duchenne
• Anatomical therapeutic chemical (ATC) code:
  M09AX03

Pharmacotherapeutic group

Other drugs for disorders of the musculo-skeletal system

Therapeutic indication

Translarna is indicated for the treatment of Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene, in ambulatory patients aged 2 years and older. Efficacy has not been demonstrated in non-ambulatory patients.

The presence of a nonsense mutation in the dystrophin gene should be determined by genetic testing.

Authorisation details

• EMA product number:
  EMEA/H/C/002720
• Conditional approval:

  This medicine received a conditional marketing authorisation. This was granted in the interest of public health because the medicine addresses an unmet medical need and the benefit of immediate availability outweighs the risk from less comprehensive data than normally required. For more information, see Conditional marketing authorisation.

• Marketing authorisation holder:
  PTC Therapeutics International Limited

  Unit 1; 52-55 Sir John Rogerson's Quay; Dublin 2; D02 NA07; Ireland

• Opinion adopted:
  23/01/2014
• Marketing authorisation issued:
  31/07/2014
• Expiry of marketing authorisation:
  28/03/2025
• Revision:
  25

Assessment history

Changes since initial authorisation of medicine

Translarna : EPAR - Procedural steps taken and scientific information after authorisation

Translarna-H-C-2720-R-0071 : EPAR - Assessment Report - Non-renewal

Adopted Reference Number: EMA/CHMP/555483/2024

Translarna-H-C-002720-II-0074 : EPAR - Assessment report - Variation

Adopted Reference Number: EMA/CHMP/116346/2024

Translarna-H-C-2720-P46-028 : EPAR - Assessment Report

Adopted Reference Number: EMA/CHMP/597049/2021

Translarna-H-C-2720-II-0047 : EPAR - Assessment Report - Variation

Adopted Reference Number: EMA/616402/2019

Translarna-H-C-2720-P46-0025 : EPAR - Assessment Report

Adopted Reference Number: EMA/649891/2019

Questions and answers on the refusal of a change to the marketing authorisation for Translarna (ataluren)

Adopted Reference Number: EMA/565599/2019

Translarna-H-C-2720-II-0049 : EPAR - Assessment Report - Variation

Adopted Reference Number: EMA/CHMP/159564/2019

Translarna-H-C-2720-II-0037 : EPAR - Assessment report - Variation

Adopted Reference Number: EMA/431114/2018

CHMP post-authorisation summary of positive opinion for Translarna (II-37)

Adopted Reference Number: EMA/304289/2018

Translarna-H-C-2720-R-0022 : EPAR - Assessment Report - Renewal

Adopted Reference Number: EMA/CHMP/738638/2016

Initial marketing authorisation documents

Translarna : EPAR - Public assessment report

Adopted Reference Number: EMA/369266/2014

CHMP summary of positive opinion for Translarna

Adopted Reference Number: EMEA/CHMP/310130/2014