European Commission’s Reconsideration of Translarna’s Marketing Authorization: A Critical Turning Point for Duchenne Muscular Dystrophy Treatment

European Commission's Decision:
The European Commission has returned the Committee for Medicinal Products for Human Use’s (CHMP) negative opinion on Translarna to the CHMP for re-evaluation, allowing the drug to remain on the market in Europe for now.

CHMP's Repeated Negative Opinions:
The CHMP has issued multiple negative opinions on Translarna, citing that the drug's effectiveness has not been confirmed in patients with nonsense mutation Duchenne muscular dystrophy.

Additional Data Consideration:
The European Commission has asked the CHMP to further consider additional real-world data and patient registry information in their re-evaluation.

Regulatory Background:
Translarna was granted a conditional marketing authorization in 2014, with the requirement for additional studies to confirm its benefits. However, subsequent studies have failed to demonstrate the drug's effectiveness, leading to repeated negative opinions from the CHMP.

Patient Advocacy Impact:
The European Commission's decision to maintain Translarna's marketing authorization despite the CHMP's negative opinions may reflect the influence of patient advocacy groups and the high unmet medical need for effective treatments for Duchenne muscular dystrophy.

US Regulatory Status:
PTC Therapeutics plans to re-submit a New Drug Application (NDA) for Translarna to the FDA by mid-2024, following feedback from the agency.