PTC Therapeutics (PTCT)

PTC Therapeutics confirms CHMP recommendation for a renewal of Translarna's Marketing Authorization for Nonsense Mutation Duchenne Muscular Dystrophy based on continued 'positive benefit-risk' assessment:

Translarna is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation.

• In connection with the renewal, the marketing authorization will include a specific obligation to conduct an additional long-term post-authorization trial.
• The CHMP has requested that PTC conduct a new 18-month randomized, placebo-controlled study in patients with nonsense mutation Duchenne muscular dystrophy, as a specific post-authorization obligation, with results expected to be available in the first quarter of 2021. This study will be followed by an 18-month open-label extension period where all patients will be switched to Translarna. PTC has proposed a trial similar in size to ACT DMD and details of the protocol are expected to be finalized in future interactions with the EMA. Conditional marketing authorizations are subject to annual reassessment and renewal.