Hailshadow
PTC Therapeutics (NASDAQ:PTCT) said it has submitted a briefing document to support renewal of its conditional approval for Translarna in Europe and plans to meet with the FDA in Q1 2024 to discuss the potential filing of a market application for US approval.
The biotech company said it submitted the briefing document to EU regulators as part of a reexamination of a negative opinion on the drug by the Committee for Medicinal Products for Human Use, or CHMP. The reexamination is expected to last until late January, after which CHMP will provide an opinion. The opinion would then be sent within 67 days for adoption by the European Commission.
PTC also met with the FDA, which suggested it request a meeting to discuss specific data and information that could be used to support a New Drug Application for the product. PTC said it expects the FDA meeting to take place in Q1 2024.
Translarna, also known as ataluren, has conditional approval in the EU for the treatment of a certain type of Duchenne muscular dystrophy. The product is not yet approved in the US.