Roche Slammed With Negative EMA CHMP Opinion for Sarepta’s DMD Gene Therapy Elevidys
The opinion recommends against conditional marketing authorization for patients who are ambulatory.
The European Medicines Agency (EMA)’s Committee for Medicinal Products for Human Use (CHMP) has issued an opinion recommending against conditional marketing authorization for Sarepta Therapeutics’ Duchenne muscular dystrophy (DMD) adeno-associated virus (AAV) vector-based gene therapy delandistrogene moxeparvovec-rokl (marketed as Elevidys) for patients who are ambulatory, according to Roche, which has commercial rights for the gene therapy product in markets outside the United States.1,2
The CHMP’s opinion was based on data from clinical trials for Elevidys, including the pivotal phase 3 EMBARK clinical trial (NCT05096221). According to Roche, sustained stabilization or slowing of disease progression was observed in patients who were treated with Elevidys in EMBARK. Roche noted that the trial’s primary end point was not met, but stated that statistically significant and clinically meaningful improvements compared to placebo were seen on functional outcome measures that the company referred to as “important secondary end points.” With regard to safety, it was noted that ambulatory patients in the trial showed a consistent and manageable safety profile.
"We are disappointed by the CHMP’s negative opinion, given the urgent need for disease-modifying therapies for children in the European Union living with Duchenne," Levi Garraway, MD, PhD, the chief medical officer and head of global product development at Roche, said in a statement.1 "With an average life expectancy of only 28 years, achieving disease stabilization is a major advance for individuals living with Duchenne, their families and caregivers. We are confident in the value Elevidys can bring to ambulatory patients."
Roche has stated its intent to continue to speak with the EMA with the goal of finding a means to bring Elevidys towards authorization in the EU. Notably, Elevidys and some of Sarepta’s other muscular dystrophy gene therapies have experienced a substantial amount of turbulence following the recent deaths of several patients. Earlier this month, the FDA had informally requested that Sarepta put shipment of all Elevidys product in the United States on hold, a request that Sarepta initially resisted but later complied with.3-6 After the FDA concluded its investigation of the death an 8-year-old boy who was treated with Elevidys in Brazil, having determined that the death was unrelated to Elevidys itself, the FDA recommended that Sarepta resume shipment of Elevidys for patients who are ambulant.
Notably, Sarepta had previously paused shipments to patients who are nonambulant. This pause, which remains in effect, was made after 2 nonambulant patients who were treated with Elevidys died in separate incidents earlier this year.5 Both of these patients’ deaths were attributed to acute liver failure.6,7
“The FDA will continue to work with the sponsor regarding nonambulatory patients, which remains subject to a voluntary hold, following 2 deaths,” the FDA wrote in its press release recommending the hold for ambulatory patients be lifted.3 “The patient community is an important voice, and the FDA will continue to listen to and respond to thoughts from the community impacted by DMD.”
The FDA also recently revoked the platform technology designation it had previously granted to Sarepta for the AAVrh74 viral vector that is used in Elevidys and several other of the company’s muscular dystrophy gene therapy products following the death of a 51-year-old patient treated with the company’s investigational gene therapy SRP-9004 for nonambulant limb-girdle muscular dystrophy (LGMD) type 2D/R3.8-10 The FDA also simultaneously placed holds on all trials currently evaluating SRP-9004 and bidridistrogene xeboparvovec (also known as SRP-9003), Sarepta’s investigational gene therapy product for LGMD type 2E.
REFERENCES
1. Roche provides regulatory update on Elevidys™ gene therapy for Duchenne muscular dystrophy in the EU. News release. Roche. July 24, 2025. Accessed July 31, 2025. https://www.roche.com/media/releases/med-cor-2025-07-25
2. DMD gene therapy Elevidys dealt another blow, this time from EU panel. News article. Matthew Dennis. FirstWord PHARMA. July 25, 2025. Accessed July 31, 2025. https://firstwordpharma.com/story/5983866
3. FDA recommends removal of voluntary hold for Elevidys for ambulatory patients. News release. FDA. July 28, 2025. Accessed July 31, 2025. https://www.fda.gov/news-events/press-announcements/fda-recommends-removal-voluntary-hold-elevidys-ambulatory-patients
4. FDA investigating death of 8-year-old boy who received Elevidys. News release. FDA. July 25, 2025. Accessed July 31, 2025. https://www.fda.gov/news-events/press-announcements/fda-investigating-death-8-year-old-boy-who-received-elevidys
5. Sarepta Therapeutics provides clarifying statement on Elevidys. News release. Sarepta Therapeutics, Inc. July 25, 2025. Accessed July 31, 2025. https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-provides-clarifying-statement-elevidys
6. Sarepta Therapeutics provides statement on Elevidys. News release. Sarepta Therapeutics, Inc. July 18, 2025. Accessed July 31, 2025. https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-provides-statement-elevidys
7. FDA informs sarepta that it recommends that sarepta remove its pause and resume shipments of ELEVIDYS for ambulatory individuals with duchenne muscular dystrophy. News release. Sarepta Therapeutics, Inc. July 28, 2025. Accessed July 31, 2025. https://investorrelations.sarepta.com/news-releases/news-release-details/fda-informs-sarepta-it-recommends-sarepta-remove-its-pause-and
8. Philippidis A. StockWatch: Sarepta shares nosedive after LGMD gene therapy patient dies. Genetic Engineering & Biotechnology News. July 20, 2025. Accessed July 31, 2025. https://www.genengnews.com/topics/genome-editing/stockwatch-sarepta-shares-nosedive-after-lgmd-gene-therapy-patient-dies/
9. FDA requests Sarepta Therapeutics suspend distribution of Elevidys and places clinical trials on hold for multiple gene therapy products following 3 deaths. News release. FDA. July 18, 2025. Accessed July 31, 2025. https://www.fda.gov/news-events/press-announcements/fda-requests-sarepta-therapeutics-suspend-distribution-elevidys-and-places-clinical-trials-hold
10. U.S. FDA grants platform technology designation to the viral vector used in SRP-9003, Sarepta’s investigational gene therapy for the treatment of limb girdle muscular dystrophy type 2E/R4. News release. Sarepta Therapeutics Inc. June 4, 2025. Accessed July 31, 2025. https://investorrelations.sarepta.com/news-releases/news-release-details/us-fda-grants-platform-technology-designation-viral-vector-used
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