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news.The European Medicines Agency (EMA) has validated the submission of a variation for a new indication for PTC Therapeutics’ Translarna (ataluren) to treat nonsense mutation cystic fibrosis (nmCF).
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The company said the variant is for the 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.
In a Phase 3 double-blind, placebo-controlled study, PTC compared Translarna to placebo in nmCF patients.
The company is undertaking an additional Phase 3 study of Translarna in nmCF patients and expects top-line data by the end of next year.
PTC Therapeutics CEO Stuart Peltz said: "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 is a protein restoration therapy designed to allow the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation.
It has the potential to benefit patients across an array of genetic disorders caused by a nonsense mutation.
Translarna, which is an investigational new drug in the US, is licensed in the European Economic Area to treat nonsense mutation Duchenne muscular dystrophy in ambulatory patients aged five years and older.
PTC secured orphan drug status from the FDA and the European Commission for Translarna to treat nonsense mutation cystic fibrosis and nonsense mutation Duchenne muscular dystrophy.