PTC Therapeutics provides regulatory update for Translarna in Europe, the U.S.

PTC Therapeutics provided an update on Translarna regulatory activities in Europe and the United States. PTC has submitted the briefing document as part of the re-examination of the initial Committee for Medicinal Products for Human Use – CHMP – negative opinion on the renewal of the conditional marketing authorization of Translarna in Europe. The re-examination procedure will focus only on the renewal of the existing conditional marketing authorization. The procedure is expected to last until late January 2024, when an opinion will be provided by CHMP. This opinion will then be sent for adoption by the European Commission, within the following 67 days. In the U.S., PTC recently participated in a Type C meeting with the FDA to discuss a potential path to a resubmission of a New Drug Application for Translarna. Based on the discussion, FDA suggested that PTC request a follow-up meeting to align on the specific contents that could support a potential NDA filing. PTC expects this meeting will occur in 1Q24. Translarna, discovered and developed by PTC Therapeutics, is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation.