Roche provides regulatory update on Elevidys™ gene therapy for Duchenne muscular dystrophy in the EU
Roche (OTCQX:RHHBY) announced that the European Medicines Agency's CHMP has issued a negative opinion on the conditional marketing authorization for Elevidys™ (delandistrogene moxeparvovec) in the EU. The therapy was intended for ambulatory individuals aged 3-7 years with Duchenne muscular dystrophy (DMD).
Despite the Phase III EMBARK study showing sustained disease stabilization and clinically meaningful improvements across secondary endpoints, the primary endpoint was not met after one year. The therapy has treated over 900 individuals with DMD, including 760 ambulatory patients, in clinical and real-world settings. Roche plans to continue dialogue with EMA to explore potential paths forward.
Roche (OTCQX:RHHBY) ha annunciato che il CHMP dell'Agenzia Europea per i Medicinali ha espresso un parere negativo sulla autorizzazione condizionata alla commercializzazione di Elevidys™ (delandistrogene moxeparvovec) nell'UE. La terapia era destinata a individui ambulanti di età compresa tra 3 e 7 anni affetti da distrofia muscolare di Duchenne (DMD).
Nonostante lo studio di Fase III EMBARK abbia mostrato una stabilizzazione duratura della malattia e miglioramenti clinicamente significativi negli endpoint secondari, l'endpoint primario non è stato raggiunto dopo un anno. La terapia ha trattato oltre 900 persone con DMD, inclusi 760 pazienti ambulanti, sia in ambito clinico che nella pratica reale. Roche intende proseguire il dialogo con l'EMA per esplorare possibili strade future.
Roche (OTCQX:RHHBY) anunció que el CHMP de la Agencia Europea de Medicamentos emitió una opinión negativa sobre la autorización condicional de comercialización de Elevidys™ (delandistrogene moxeparvovec) en la UE. La terapia estaba destinada a personas ambulatorias de 3 a 7 años con distrofia muscular de Duchenne (DMD).
A pesar de que el estudio de Fase III EMBARK mostró una estabilización sostenida de la enfermedad y mejoras clínicamente significativas en los puntos finales secundarios, no se cumplió el punto final primario tras un año. La terapia ha tratado a más de 900 personas con DMD, incluyendo 760 pacientes ambulatorios, en entornos clínicos y en la práctica real. Roche planea continuar el diálogo con la EMA para explorar posibles vías a seguir.
로슈(OTCQX:RHHBY)는 유럽 의약품청(EMA)의 CHMP가 EU 내 Elevidys™(델란드스트로진 모세파르보벡)의 조건부 시판 허가에 대해 부정적인 의견을 발표했다고 밝혔습니다. 이 치료법은 듀센 근이영양증(DMD) 환자 중 3~7세 보행 가능한 환자를 대상으로 했습니다.
3상 EMBARK 연구에서 질병의 지속적인 안정화와 2차 평가변수에서 임상적으로 의미 있는 개선이 나타났음에도 불구하고, 1년 후 1차 평가변수는 충족하지 못했습니다. 이 치료법은 임상 및 실제 환경에서 900명 이상의 DMD 환자, 그중 760명의 보행 가능한 환자를 치료했습니다. 로슈는 EMA와의 대화를 계속하며 향후 가능성을 모색할 계획입니다.
Roche (OTCQX:RHHBY) a annoncé que le CHMP de l'Agence européenne des médicaments a émis un avis négatif concernant l'autorisation de mise sur le marché conditionnelle d'Elevidys™ (delandistrogene moxeparvovec) dans l'UE. Ce traitement était destiné aux patients ambulatoires âgés de 3 à 7 ans atteints de dystrophie musculaire de Duchenne (DMD).
Bien que l'étude de phase III EMBARK ait montré une stabilisation durable de la maladie et des améliorations cliniquement significatives sur les critères secondaires, le critère principal n'a pas été atteint après un an. Le traitement a été administré à plus de 900 patients atteints de DMD, dont 760 patients ambulatoires, en milieu clinique et en conditions réelles. Roche prévoit de poursuivre le dialogue avec l'EMA pour explorer les voies possibles à suivre.
Roche (OTCQX:RHHBY) gab bekannt, dass der CHMP der Europäischen Arzneimittelagentur eine negative Stellungnahme zur bedingten Marktzulassung von Elevidys™ (delandistrogene moxeparvovec) in der EU abgegeben hat. Die Therapie war für ambulante Patienten im Alter von 3 bis 7 Jahren mit Duchenne-Muskeldystrophie (DMD) vorgesehen.
Obwohl die Phase-III-EMBARK-Studie eine anhaltende Stabilisierung der Erkrankung und klinisch bedeutsame Verbesserungen bei den sekundären Endpunkten zeigte, wurde der primäre Endpunkt nach einem Jahr nicht erreicht. Die Therapie wurde bei über 900 Personen mit DMD angewendet, darunter 760 ambulante Patienten, sowohl in klinischen Studien als auch im realen Einsatz. Roche plant, den Dialog mit der EMA fortzusetzen, um mögliche Wege für die Zukunft zu erkunden.
- None.
- CHMP issued negative opinion for EU marketing authorization
- Failed to meet primary endpoint in Phase III EMBARK study after one year
- Limited treatment window (only for ambulatory patients aged 3-7 years)
- Regulatory setback may delay access for EU patients
- EMA’s CHMP issued an opinion not to recommend Elevidys™ (delandistrogene moxeparvovec) for the treatment of ambulatory individuals with Duchenne muscular dystrophy (DMD)
- Roche will continue its dialogue with the EMA to explore a potential path forward to make Elevidys available to individuals living with DMD in the EU
- Roche believes the benefit-risk remains positive in the ambulatory Duchenne population
- Elevidys is the first and only disease-modifying gene therapy for DMD
Basel, 25 July 2025 - Roche (SIX: RO, ROG; OTCQX: RHHBY) 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). Given the high unmet need in DMD, Roche plans to continue to work with the EMA to explore a potential path forward.
"We are disappointed by the CHMP’s negative opinion, given the urgent need for disease-modifying therapies for children in the EU living with Duchenne," said Levi Garraway, M.D., Ph.D., Chief Medical Officer and Head of Global Product Development, Roche. "With an average life expectancy of only 28 years, achieving disease stabilisation 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."
The CHMP opinion is based on data from the largest and broadest gene therapy clinical programme in DMD to date, including results from the pivotal Phase III EMBARK study that showed treatment with Elevidys provided sustained stabilisation or slowing of disease progression, and a consistent and manageable safety profile in ambulatory patients. To date, more than 900 individuals with DMD, 760 of whom are ambulatory, have been treated with Elevidys in clinical and real-world settings.
While the primary endpoint was not met in EMBARK after one year, Elevidys showed clinically meaningful and statistically significant improvements across important secondary endpoints of functional outcome measures when compared to placebo. Longer term efficacy data were also submitted to EMA, including two-year results from the EMBARK study and three-year pooled efficacy analysis from three other Elevidys studies that showed clinically meaningful improvements across key measures of motor function. One-year data from part one of the EMBARK study were published in Nature Medicine in October 2024 and results from year two were shared at this year’s Muscular Dystrophy Association clinical & scientific conference in Dallas, TX.
DMD is a rare, genetic, muscle-wasting disease that progresses rapidly from early childhood. Everyone with Duchenne will eventually lose the ability to walk, along with upper limb, lung, and cardiac function. Average life expectancy is only 28 years. The physical, emotional, and financial impact of Duchenne on those affected, their families, and caregivers, is profound. Roche recognises there is a significant unmet medical need for those living with Duchenne and the urgency for treating children before DMD progresses to provide the best possible chance for improved outcomes. We are actively working with health authorities across the globe to bring Elevidys to patients and their families as soon as possible.
Elevidys is the first and only approved gene therapy targeting the underlying cause of disease that consistently demonstrates stabilisation or slowing of DMD disease progression, with durable effects on functional and biological outcomes and muscle health.
About Duchenne muscular dystrophy
DMD primarily affects males, with 1 in 5,000 boys born worldwide having Duchenne. Everyone with Duchenne will eventually lose the ability to walk, along with upper limb, lung and cardiac function. Average life expectancy is only 28 years. The physical, emotional and financial impact of Duchenne on those affected, their families and caregivers, is profound.
Duchenne is an X-linked, rare neuromuscular disease caused by pathogenic variants (mutations) in the DMD gene that disrupt the production of functional dystrophin protein, leading to progressive and irreversible muscle weakness, diminished quality of life and premature death. Dystrophin strengthens and protects muscles and without it, normal activity causes excessive damage to muscle cells as they are more sensitive to injury. Over time, muscle tissue is replaced with scar tissue and fat, causing muscles to weaken. Although Duchenne progresses differently in each individual, its devastating trajectory is well established. Those with Duchenne will eventually lose the ability to use and move their limbs, to breathe on their own and are susceptible to respiratory infections. Muscle damage to the heart causes cardiomyopathy, including rhythm abnormalities and heart failure.
Early diagnosis is important for timely intervention to prolong muscle function and preserve quality of life. There is a critical need for disease-modifying treatments that address the underlying cause of DMD before irreversible muscle loss occurs.
About Elevidys™ (delandistrogene moxeparvovec)
Elevidys™ (delandistrogene moxeparvovec, also known as SRP-9001) is the first approved disease-modifying gene therapy for Duchenne and is designed to address the underlying cause of Duchenne through targeted skeletal, respiratory and cardiac muscle expression of shortened dystrophin produced by Elevidys. Elevidys is a one-time treatment administered through a single intravenous dose. Elevidys is contraindicated in individuals with any deletion in exons 8 and/or 9 in the DMD gene.
Elevidys has been studied in the largest and broadest gene therapy clinical development program in DMD with the longest follow-up (up to six years) in patients of all ages, and with various DMD mutations. To date, more than 900 individuals with DMD (more than 760 of whom are ambulatory) have been treated with Elevidys across clinical and real-world settings and Elevidys is now approved in nine countries, including Japan.
Important Elevidys Updates:
On 15 June, Roche announced new dosing restrictions for Elevidys for non-ambulatory DMD patients, irrespective of age, in both clinical and commercial settings. These measures followed two reported fatalities in the non-ambulatory DMD population. On 22 July, in response to the U.S. Food and Drug Administration (FDA)’s request to Sarepta, Roche took additional measures towards initiating a voluntary and temporary pause of any new orders of Elevidys to countries outside the U.S. that reference the FDA as the basis for their local approval, and in Named Patient Supply (NPS) countries. Discussions with other relevant health authorities are ongoing. Roche will immediately respect requests to halt new orders and shipments from health authorities.
Patient safety is Roche’s highest priority. Based on the totality of available data, Roche believes that the benefit-risk profile is positive in the ambulatory patient population. To date, approximately 760 ambulatory DMD patients have been treated with Elevidys in clinical and real-world settings and there have been no treatment-related fatalities.
Elevidys is being developed by Roche in collaboration with Sarepta Therapeutics
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Neuroscience is a major focus of research and development at Roche. Our goal is to pursue groundbreaking science to develop new treatments that help improve the lives of people with chronic and potentially devastating diseases.
Roche is investigating more than a dozen medicines for neurological disorders, including multiple sclerosis, spinal muscular atrophy, neuromyelitis optica spectrum disorder, Alzheimer’s disease, Huntington’s disease, Parkinson’s disease and Duchenne muscular dystrophy. Together with our partners, we are committed to pushing the boundaries of scientific understanding to solve some of the most difficult challenges in neuroscience today.
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