PTC submits EU application for Translarna for cystic fibrosis

The European Medicines Agency (EMA) has validated the submission of a variation for a new indication for PTC Therapeutics’ Translarna (ataluren) for the treatment of nonsense mutation cystic fibrosis (nmCF) for patients not taking chronic inhaled aminoglycoside antibiotics.

If approved, Translarna would be the first oral protein restoration treatment that targets the underlying cause of nmCF. Approximately 10% of cystic fibrosis patients have their disease as a result of a nonsense mutation, which can cause the most severe form of cystic fibrosis. 

“We are very excited to be advancing our second indication, which represents another milestone in realising the full potential for Translarna,” said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics. “We are eager to bring Translarna to patients with nonsense mutation cystic fibrosis, who currently have no other treatment options for the underlying cause of their disorder. We look forward to working with regulators to help bring this precision based medicine to patients as quickly as possible.”

Translarna enables formation of a functioning protein in genetic disorders caused by a nonsense mutation

“Nonsense mutation cystic fibrosis is a serious form of cystic fibrosis and is very challenging to treat. We are pleased that there may be a treatment option for people with nmCF in the near future,” said Jacquelien Noordhoek, President of CF Europe. “The progress that is being made to better understand and treat the underlying genetic causes of cystic fibrosis is critical and must continue.”

The regulatory application for Translarna for nmCF is based on previously announced analyses from the Company’s completed Phase 3 double-blind, placebo-controlled study comparing Translarna to placebo in nmCF patients. PTC is conducting an additional randomised, double-blind, placebo-controlled Phase 3 study of Translarna in nmCF patients and expects enrollment to be completed by the end of this year, with top-line data expected by the end of 2016.

Translarna 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.