DMD: extending the indication for boys aged 2 to 5

Translarna (ataluren) is the first drug to have obtained a conditional marketing authorization in Europe, in Duchenne muscular dystrophy:

  • On July 31, 2014, the conditional marketing authorization was issued by the EMA only for children with DMD aged 5 years and over, walking and having a nonsense mutation in the DMD gene.
  • On May 31, 2018, the EMA extends the indication to children aged 2 to 5 years old, walking.
    The EMA is also renewing the conditional marketing authorization until September 2022 and is asking the PTC Therapeutics laboratory to obtain convincing efficacy results by then. Translarna studies are underway for this purpose (long-term open-label phase III trial, and European STRIDE register).
  • On October 23, 2019 the Haute Autorité de Santé (HAS, the french health agency) issued an opinion in which it considers that this medicine has a weakened medical service in boys with DMD from the age of 2 years, still walking, while specifying that there is no improvement in the medical service rendered (ASMR V) in children aged 2 to 5 years.

To date, the Translarna is issued under the post-ATU (Temporary Authorization of Use) device, at the hospital and is temporarily supported, but only for patients over 5 years of age. For all its indications, the final negotiations of price and conditions of reimbursement of the product are in progress.

 

Read the HAS notice (2019, french) from the Commisson de transparence de l’HAS

 

Read the EMA notice (2018) An overview of Translarna and why it is authorised in the EU