The European Union’s health regulatory agency did not endorse approving Elevidys for ambulatory patients with Duchenne muscular dystrophy.
The European Union’s Committee for Medicinal Products for Human Use declined to endorse the use of the gene therapy Elevidys for ambulatory patients with Duchenne muscular dystrophy.
The news was announced Friday morning by Roche, which owns rights to Sarepta Therapeutics’ Elevidys in the region. The pharma said it will continue working with the European Medicines Agency (EMA)—the regulatory body under which the CHMP falls—to figure out a way forward for Elevidys, citing the “high unmet need in DMD.”
In a note to investors on Friday, analysts at William Blair said that the negative opinion was “unfortunate but unsurprising,” delivering another hit to Sarepta due to “reduced revenue from potential future milestone and royalty payments from Roche.”
Roche, which paid Sarepta $1.15 billion upfront in December 2019 for ex-U.S. rights to the asset, submitted for EU approval of Elevidys for ambulatory patients aged 3 to 7 years in June 2024. The pharma was awaiting CHMP’s opinion as Elevidys became embroiled in a safety scandal following multiple patient deaths and Sarepta’s subsequent handling of the situation.
In March, the Massachusetts-based biotech reported that one non-ambulatory patient with DMD had died from acute liver failure after being treated with Elevidys. This complication is a known side-effect of gene therapies delivered using adeno-associated virus (AAV) vectors. But after another non-ambulatory patient died in June, Sarepta suspended shipments to non-ambulatory patients. Then at a corporate update last week, the company announced it was adding a black box warning to the therapy.
The very next day, however, news broke of a third patient death, this time in a patient dosed with the investigational gene therapy SPR-9004, being studied for limb-girdle muscular dystrophy but which uses the same vector technology. In light of this third mortality, the FDA asked the company to suspend all Elevidys shipments. After some resistance, Sarepta on Monday gave in to the request.
Roche then followed Sarepta’s lead and temporarily halted deliveries to non-ambulatory patients in countries that have based their domestic approvals based on the FDA’s regulatory decisions. The pharma held strong on shipping to the ambulatory population, however. “Roche believes that the benefit-risk profile is positive,” a spokesperson for the pharma told BioSpace over email on Wednesday, citing “the totality of available data.”
“We are disappointed by the CHMP’s negative opinion,” Roche chief medical officer Levi Garraway said in a prepared statement on Friday, pointing out the “urgent need for disease-modifying therapies for children in the EU living with Duchenne.”
Neither the CHMP nor the EMA have the authority to approve or decline Roche’s marketing authorization application for Elevidys in the European Union. The bodies will instead pass the assessment onto the European Commission, which should then issue a legally binding decision within 67 days.
