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

– European Commission ratification anticipated in coming months –
– Approval of the Translarna annual re-assessment also recommended by CHMP –

PTC Therapeutics today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has 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. This recommendation is based on the CHMP’s review of PTC’s Study 030 trial results which the CHMP concluded demonstrates a positive benefit-risk ratio in this population. In addition to the label expansion, the CHMP has also recommended the renewal of the current marketing authorisation 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, 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 2 years of age is planned to start immediately at the time of EC ratification.

About ataluren (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. Translarna, tradename of ataluren, is licensed in the European Economic Area for the treatment of nonsense mutation Duchenne muscular dystrophy in ambulatory patients aged five years and older. Ataluren is an investigational new drug in the United States.

PTC Therapeutics Press Release