PTC Therapeutics gets EMA validation for variant of cystic fibrosis drug
PTC Therapeutics (Nasdaq: PTCT) on Wednesday said the European Medicines Agency has validated the submission for a variant of Translarna (ataluren) to treat nonsense mutation cystic fibrosis (nmCF).
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. About 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.
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