Positive news for Translarna™ (ataluren)


One of the objectives of our team is to inform you about new treatments. Here’s the latest news from PTC Therapeutics press release about Translarna ™ (ataluren). Enjoy reading and thank you for sharing these articles within the DMD community.


CHMP Adopts Positive Opinion for the Expansion of the Translarna ™ (ataluren) Label to Include Patients as Young as 2 Years of Age

PTC Therapeutics, Inc. announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) had recommended approval of expanding the indication of Translarna ™ (ataluren) to include ambulatory children aged two to five years with nonsense mutation Duchenne muscular dystrophy (nmDMD). This broadens the use beyond the current indication which is for ambulatory patients who are over five years of age. In addition to the label expansion, the CHMP has also recommended the renewal of the current marketing authorization of Translarna.

“Early diagnosis and treatment has been a paramount part of our strategy and this recommendation perfectly aligns with our vision of giving best-in-class treatment to patients,” said Marcio Souza, the chief operating officer of PTC Therapeutics.

PTC’s focus on early patient identification and market readiness have been intensified in anticipation of the CHMP recommendation and the launch of Translarna for patients as young as two years of age, and it is planned to start immediately at the time of EC ratification.


About  Translarna ™ (ataluren)

Discovered and developed by PTC Therapeutics, Inc., Translarna ™ (ataluren) is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation. A nonsense mutation is an alteration in the genetic code that prematurely halts the synthesis of an essential protein. The resulting disorder is determined by which protein cannot be expressed in its entirety and is no longer functional, such as dystrophin in Duchenne muscular dystrophy.

read more: https://laforcedmd.com/ataluren-promising-treatment-for-dmd/


About DMD

Duchenne muscular dystrophy (DMD) is a disease that almost exclusively affects boys and whose incidence is 1 in 3,500. It is extremely rare that Duchenne muscular dystrophy (DMD) will affect girls. Those affected are usually diagnosed around the age of five, but symptoms may be visible from early childhood. It is a degenerative disease of the muscles caused by a genetic mutation. The Duchenne muscular dystrophy (DMD) – for which no treatment is currently available – directly affects skeletal muscles. Without treatment, the consequences of the disease are dire for those afflicted and their families.


Press release:  http://ir.ptcbio.com/news-releases/news-release-details/chmp-adopts-positive-opinion-expansion-translarnatm-ataluren

3 replies
  1. boudiaf mousaab
    boudiaf mousaab says:

    My 3 year old son is suffering from dystrophy muscular duchenne (DMD)
    Can he take this medicine?

    • Marie-Catherine Du Berger
      Marie-Catherine Du Berger says:

      “Ataluren is in clinical development for the treatment of Duchenne muscular dystrophy caused by a nonsense mutation (nmDMD). Ataluren is approved within the European Union (EU) Member States, Iceland, Liechtenstein, Norway, Israel and South Korea under the trade name Translarna™ for the treatment of nmDMD in ambulatory patients aged five years and older. ” – https://www.ptcbio.com/en/pipeline/ataluren-translarna/ Your doctor is the best person for answering your questions.


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