Duchenne drug Translarna approved in Russia

USA-based rare disease drug developer PTC Therapeutics’ (Nasdaq: PTCT) Translarna (ataluren) has been granted marketing approval in the Russian Federation for nonsense mutation Duchenne muscular dystrophy (nmDMD).

It is estimated that 13% of DMD patients have a nonsense mutation. In countries where it is approved, Translarna is currently the only medicine that targets the underlying cause of nmDMD.1

"Translarna was the first therapeutic ever approved for nonsense mutation Duchenne muscular dystrophy patients and we are excited to expand approval of Translarna into Russia," stated Stuart Peltz, chief executive of PTC Therapeutics, adding: "There has been great interest from Russian physicians to be able to access a treatment that targets the underlying cause of the disease, and a number of nmDMD patients have already been identified who can potentially benefit from Translarna. Our goal is to bring this product to nmDMD patients globally and this approval marks another important milestone."