Roche announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) issued a negative opinion on the conditional marketing authorisation (CMA) for Elevidys (delandistrogene moxeparvovec) for ambulatory individuals aged three to seven years with Duchenne muscular dystrophy (DMD). – Read More –
Given the high unmet need in DMD, Roche plans to continue to work with the EMA to explore a potential path forward.
Why Elevidys Was Not Approved by the European Medicines Agency (EMA)
Key reasons for refusal
According to the EMA’s public assessment specific reasons include:
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- In the pivotal study (the main evidence submitted), there was no statistically significant improvement in motor‑function (as measured using the North Star Ambulatory Assessment, NSAA) at 12 months between the Elevidys arm and placebo arm. The difference was only 0.65 points on a 34‑point scale, which the EMA concluded “may be due to chance”.
- Although the therapy induced expression of a shorter form of the dystrophin protein, the levels of that dystrophin expression could not be reliably linked to meaningful improvements in movement ability.
- Even when considering sub‑groups of patients who appeared to respond better, the company could not convincingly show that the treatment provided a clinically meaningful benefit in those groups either.
- Because the company had applied for a conditional marketing authorisation (CMA), the standard the EMA applies is that there must be a positive benefit‑risk (i.e., evidence of benefit + manageable risk) with some compelling data to support it. The EMA concluded that the benefits have not been sufficiently demonstrated in this context, and so refused the authorisation.
Learn More: Roche Suspends Shipments of Elevidys Gene Therapy Outside of the United States
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