PTC's Translarna OK'd in Russia for Duchenne

 

• PTC Therapeutics (PTCT -1.5%) announces the regulatory nod in Russia for Translarna (ataluren) for nonsense mutation Duchenne muscular dystrophy (DMD) in patients at least two years old.
• The company has experienced headwinds getting the product approved in major markets. The FDA is not yet on board sending the company a CRL three years ago. The European Medicines Agency conditionally approved it in 2014 for ambulatory DMD patients at least five years old (later expanded to patients at least two years old) but it rejected its application for non-ambulatory patients last year. It withdrew its European application for cystic fibrosis in 2017. Brazil's National Health Surveillance Agency approved it in 2019 for ambulatory DMD patients at least five years old.

https://seekingalpha.com/news/3641734-ptcs-translarna-okd-in-russia-for-duchenne-muscular-dystrophy