Source: European Parliament
In September 2023, the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) recommended to refuse the annual renewal of the conditional marketing authorisation for Translarna. The opinion was confirmed in January 2024 following a re-examination.
In May 2024 the Commission asked the CHMP to consider further data brought to its attention. The CHMP reviewed its assessment and confirmed its recommendation in June 2024 and, after a re-examination, on 17 October 2024.
The benefit-risk was re-assessed based on available data including that from the time of the conditional approval, that generated as per the specific obligations under the conditional approval (two clinical trials), that from the patient registry STRIDE, additional real-world data and from two paediatric studies. These data failed to confirm a positive benefit-risk balance.
In line with the legislation[1], the Commission shall prepare a draft decision based on the CHMP opinion and submit it to the Standing Committee on Medicinal Products for Human Use.
In a Committee meeting on 12 December 2024, Member States considered the CHMP assessment robust but recognised the unmet medical need of Duchenne muscular dystrophy (DMD) patients and the very specific circumstances of this case.
Member States discussed the need to manage therapeutic continuity for patients already under treatment given the absence of alternative treatments authorised in the EU.
The Commission is considering how these aspects can be integrated into the final Decision which Member States will vote on early in 2025.
There are currently 32 treatments for DMD under development in the EU. EMA is currently reviewing 2 such medicines: givinostat and the gene therapy delandistrogene moxeparvovec .
- [1] https://health.ec.europa.eu/medicinal-products/legal-framework-governing-medicinal-products-human-use-eu_en