US-based PTC Therapeutics has secured marketing approval in Russia for Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD).
The approval for Translarna has been given by the Ministry of Health of the Russian Federation for Duchenne muscular dystrophy caused by a nonsense mutation in patients, aged two years and older.
Stuart W. Peltz – CEO of PTC Therapeutics said: “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.
“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.”
Translarna is a protein restoration therapy that has been designed to facilitate the formation of a functioning protein in patients having genetic disorders caused by a nonsense mutation.
A nonsense mutation is defined as a modification in the genetic code that stops the synthesis of an essential protein in a premature manner. The disorder that results is determined by the type of protein that cannot be expressed in its entirety and is no longer functional, such as the dystrophin protein in Duchenne muscular dystrophy.