PTC Therapeutics has announced, on the 5th of December 2023, an update on their regulatory progress with the European Medicines Association (EMA) and US Food & Drug Association (FDA).
PTC Therapeutics has submitted a briefing document as a 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. PTC believes they can address the concerns raised by the CHMP and appeal the decision not to renew the existing marketing authorisation. A decision is expected by the end of January 2024 by the CHMP, with the updated opinion being adopted by the EMA within 67 days thereafter.
A Type C meeting was held with the US FDA to discuss a path forward for resubmission for a New Drug Application (NDA) for Translarna, considering the evidence to date collected from clinical trials and STRIDE registry. A follow up meeting with the FDA is planned for the first quarter of 2024 to align on the specific contents that could support a potential NDA filing.
Matthew B. Klein, M.D., Chief Executive Officer of PTC, stated:
“PTC continues to work to secure global registrations that will enable access to Translarna for patients with nmDMD worldwide,” further adding. “The data collected to date demonstrate the short-and long-term benefits of Translarna on key functional aspects of disease, including ambulatory and neuromuscular function. In Europe, we believe we can address the concerns raised by the CHMP in its initial negative opinion on the renewal of Translarna conditional authorization. In the U.S., we look forward to continuing to work collaboratively with FDA on the contents of a potential NDA resubmission.”
Breakdown of the technical language:
- CHMP – The EMA’s regulatory body overseeing all medical products for human consumption.
- CHMP Marketing authorisation – the designation for sale and distribution within the EU.
- STRIDE Registry – The STRIDE Registry is the first drug registry for DMD and is demonstrating the safety and effectiveness of ataluren over a long treatment exposure in routine clinical practice
- nmDMD – Nonsense-mutation Duchenne muscular dystrophy
- Type C meeting – There are three types of meetings that occur between sponsors or applicants and FDA staff: Type C meetings consider reviews of therapeutics already advanced past Phase 1 and 2 trials, and are not considered to be stalled.
To find out more about PTC Therapeutics regulatory updates please follow this link: PTC Therapeutics
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