Latest news with #Elevidys™
Business Upturn
25-07-2025
- Business
- Business Upturn
Roche provides regulatory update on Elevidys™ gene therapy for Duchenne muscular dystrophy in the EU
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 About Roche in Neuroscience 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. About Roche Founded in 1896 in Basel, Switzerland, as one of the first industrial manufacturers of branded medicines, Roche has grown into the world's largest biotechnology company and the global leader in in-vitro diagnostics. The company pursues scientific excellence to discover and develop medicines and diagnostics for improving and saving the lives of people around the world. We are a pioneer in personalised healthcare and want to further transform how healthcare is delivered to have an even greater impact. To provide the best care for each person we partner with many stakeholders and combine our strengths in Diagnostics and Pharma with data insights from the clinical practice. For over 125 years, sustainability has been an integral part of Roche's business. As a science-driven company, our greatest contribution to society is developing innovative medicines and diagnostics that help people live healthier lives. Roche is committed to the Science Based Targets initiative and the Sustainable Markets Initiative to achieve net zero by 2045. Genentech, in the United States, is a wholly owned member of the Roche Group. Roche is the majority shareholder in Chugai Pharmaceutical, Japan. For more information, please visit All trademarks used or mentioned in this release are protected by law. Roche Global Media Relations Phone: +41 61 688 8888 / e-mail: [email protected] Hans Trees, PhD Phone: +41 79 407 72 58 Sileia Urech Phone: +41 79 935 81 48 Nathalie Altermatt Phone: +41 79 771 05 25 Lorena Corfas Phone: +41 79 568 24 95 Simon Goldsborough Phone: +44 797 32 72 915 Karsten Kleine Phone: +41 79 461 86 83 Kirti Pandey Phone: +49 172 6367262 Yvette Petillon Phone: +41 79 961 92 50 Dr Rebekka Schnell Phone: +41 79 205 27 03 Roche Investor Relations Investor Relations North America Loren KalmPhone: +1 650 225 3217 e-mail: [email protected] Attachment Media Investor Release Elevidys CHMP Opinion English Disclaimer: The above press release comes to you under an arrangement with GlobeNewswire. Business Upturn takes no editorial responsibility for the same. Ahmedabad Plane Crash
Yahoo
27-01-2025
- Health
- Yahoo
Roche announces new results from EMBARK demonstrating significant sustained benefits of Elevidys in ambulatory individuals with Duchenne muscular dystrophy (DMD)
Across three key functional outcomes, North Star Ambulatory Assessment (NSAA), Time to Rise (TTR) and 10-meter walk/run (10MWR), results were statistically significant and clinically meaningful two years after treatment with Elevidys, compared to a pre-specified propensity-weighted untreated external control group Functional differences between individuals treated with Elevidys and those in the external control group increased between one year and two years after treatment No new safety signals observed further reinforcing the consistent and manageable safety profile observed with Elevidys to date Basel, 27 January 2025 - Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today positive topline results from year two of the EMBARK trial, a global, randomised, double-blind phase III study of Elevidys™ (delandistrogene moxeparvovec), the first approved gene therapy for the treatment of individuals with Duchenne muscular dystrophy. Two years after treatment with Elevidys, statistically significant and clinically meaningful improvements were observed across three key motor function measures of NSAA, TTR and 10MWR, when compared to a pre-specified propensity-weighted untreated external control group.* Functional differences between individuals treated with Elevidys and those in the external control group increased between one and two years after dosing. Together, these results demonstrate consistent, sustained benefit in favour of Elevidys. Detailed results from year two of the EMBARK study will be shared at an upcoming medical meeting and discussed with health authorities. One-year data from part one of the EMBARK study were published in Nature Medicine in October 2024. 'After two years of treatment with Elevidys, we are seeing multiple sustained benefits in the day-to-day lives of these young boys, all of which are indicators of its disease modifying potential in Duchenne,' said Levi Garraway, M.D., Ph.D., Roche's Chief Medical Officer and Head of Global Product Development. "These results, which include improvements in standing, walking and running, represent meaningful progress and we plan to share them with health authorities as quickly as possible.' Individuals treated in part one of EMBARK (n=63) showed clinically meaningful and statistically significant improvements on the NSAA were sustained two years after treatment with Elevidys. 'As Duchenne progresses, children will lose the ability to walk, have difficulty breathing, and develop heart problems, all of which severely impact their health and ability to fully participate in life,' said Professor Francesco Muntoni, Director of Dubowitz Neuromuscular Centre, Great Ormond Street Hospital for Children, UK. 'Encouraging results from year two of the EMBARK trial suggest that with innovative treatments like Elevidys, the period of mobility and independence can potentially be improved, reducing the physical and emotional challenge Duchenne poses for these young boys and their families.' For individuals treated in part one, two years after treatment, functional motor improvements include: Functional endpoints (treated in part one) Measure compared to the external control group (LSM mean difference) NSAA +2.88 points (improvement), P<0.0001 TTR -2.06 seconds (improvement), P<0.0033 10MWR -1.36 seconds (improvement), P<0.0028 Individuals who received a placebo in part one crossed over at 52 weeks and were treated with Elevidys in part two. Despite being one year older than those treated in part one, the crossover treated group (n=59) experienced similar changes 52 weeks after treatment, favouring Elevidys compared to the external control. Individuals treated in part one were between the ages of four and seven years, and in part two, individuals were between the ages of five and nine years. For individuals treated in part two, one year after treatment, functional motor improvements include: Functional endpoints (treated in part two) Measures compared to the external control (pre-therapy baseline; LSM mean difference) NSAA +2.34 points (improvement), P<0.0001 TTR -2.70 seconds (improvement), P<0.0001 10MWR -1.07 seconds (improvement), P=0.0001 Muscle biopsies from a subset of patients taken 64 weeks after dosing in part one showed consistent and sustained expression of micro-dystrophin, as measured by western blot. Muscle pathology on MRI continues to show minimal progression in underlying muscle pathology and remains highly consistent with the functional benefits shown. No new safety signals were observed, reinforcing the consistent and manageable safety profile of Elevidys to date. Elevidys is approved for people living with Duchenne aged four years old and over regardless of their ambulatory status in the US, United Arab Emirates (UAE), Qatar, Kuwait, Bahrain and Oman. Elevidys is also approved for the treatment of ambulatory individuals aged four through seven years in Brazil and Israel. Filings have also been submitted to the European Medicines Agency (EMA) and regulatory authorities in Japan, Switzerland, Singapore, Hong Kong and Saudi Arabia. In 2019, Roche entered into a global collaboration agreement with Sarepta Therapeutics, Inc. to commercialise Elevidys in territories outside the U.S. Elevidys clinical development programme Study 101 (NCT03375164), a Phase I/II study evaluating the safety of Elevidys in four ambulatory participants aged 4 to <8 years old with Duchenne. The study is complete. Study 102 (NCT03769116), a Phase II clinical trial evaluating the safety and efficacy of Elevidys in patients with Duchenne aged 4 to <8 years. The study is complete. ENDEAVOR (Study 103, NCT04626674), a two-part, open-label, Phase Ib study assessing Elevidys micro-dystrophin protein expression and safety of Elevidys in seven cohorts of boys with Duchenne, across different ages, mutations and stages of disease progression. Study 104 (NCT06241950), a Phase I open-label, systemic gene delivery study to evaluate the safety, tolerability and expression of Elevidys in association with imlifidase in individuals aged 4 to 9 years with pre-existing antibodies to recombinant adeno-associated virus serotype, rAAVrh74. HORIZON (Study 105, NCT06597656), a Phase I open-label, systemic gene delivery study to evaluate the safety, tolerability and expression of Elevidys following plasmapheresis in individuals aged 4 to 8 years with pre-existing antibodies to adeno-associated virus serotype, AAVrh74. EMBARK (Study 301, NCT05096221), a multinational, Phase III, randomised, double-blind, placebo-controlled study assessing the safety and efficacy of Elevidys in ambulatory boys aged 4 to 7 years. The study duration is two years. ENVOL (Study 302, NCT06128564), a Phase II study evaluating the safety of Elevidys and expression of Elevidys micro-dystrophin protein in young children, including babies and newborns. ENVISION (Study 303, NCT05881408), a global Phase III study investigating the safety and efficacy of Elevidys in participants who are ambulatory (aged 8 to <18 years old) and non-ambulatory (no age limitation). The study Is recruiting. EXPEDITION (Study 305, NCT05967351), a Phase III long-term five-year follow-up study evaluating the safety and efficacy of Elevidys in those who have received Elevidys in a previous clinical study. EXPEDITION is enrolling by invitation. About EMBARKEMBARK is a multinational, phase III, randomised, double-blind, two-part crossover, placebo-controlled study assessing the safety and efficacy of Elevidys in ambulatory boys with a confirmed mutation in the DMD gene, aged four to seven years at the beginning of the trial. Eligible participants received a single dose of Elevidys during either part one or part two of the study. The study is complete. Participants (n=126) received 1.33x1014 vector genomes per kilogram bodyweight (vg/kg) of Elevidys or placebo. In part one, participants were randomised according to age (4-5y or 6-7y) or NSAA total score at screening (≤22 or >22) to receive either Elevidys or placebo, with a follow-up period for 52 weeks. In part two, participants crossed over - meaning, those who were previously treated with placebo in part one received Elevidys and participants who were previously treated with Elevidys received placebo, with a follow-up period for 52 weeks. The primary endpoint of the trial was change from baseline in NSAA total score at week 52. Secondary endpoints included: The quantity of Elevidys micro-dystrophin protein expression at Week 12 as measured by western blot of biopsied muscle tissue Change from baseline to Week 52 in time to rise from floor Change from baseline to Week 52 in 10-metre walk/run (10MWR) Change from baseline to Week 52 in stride velocity 95th centile (as measured by Syde®, a wearable device) Change from baseline to Week 52 in 100-metre walk/run Change from baseline to Week 52 in time to ascend four steps NSAA is a 17-item rating scale that is used to measure functional motor abilities and monitor disease progression in ambulant children with DMD. About ELEVIDYS™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. About Duchenne muscular dystrophyDuchenne is a rare, genetic, muscle-wasting disease that progresses rapidly from early childhood. Approximately 1 in 5,000 boys worldwide are born with Duchenne, while Duchenne in girls is very rare. Everyone who has Duchenne will lose the ability to walk, upper limb, lung and cardiac function and mean life expectancy is 28 years. A diagnosis of Duchenne will require full-time caregiving which is most often provided by parents, the majority of whom will find it difficult to carry out usual work or household activities and suffer from depression and physical pain. Duchenne is caused by mutations of the DMD gene, which affects the production of the muscle protein, dystrophin. Dystrophin is a critical component of a protein complex that strengthens muscle fibers and protects them from injury during muscle contraction. Due to a genetic mutation in the DMD gene, people with Duchenne do not make functional dystrophin; their muscle cells are more sensitive to injury and muscle tissue is progressively replaced with scar tissue and fat. As dystrophin is also deficient in vital organ systems such as the cardiovascular and respiratory systems, the effect is thus inevitably fatal, with an average survival limited to the third decade of life. About Roche in NeuroscienceNeuroscience 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. About RocheFounded in 1896 in Basel, Switzerland, as one of the first industrial manufacturers of branded medicines, Roche has grown into the world's largest biotechnology company and the global leader in in-vitro diagnostics. The company pursues scientific excellence to discover and develop medicines and diagnostics for improving and saving the lives of people around the world. We are a pioneer in personalised healthcare and want to further transform how healthcare is delivered to have an even greater impact. To provide the best care for each person we partner with many stakeholders and combine our strengths in Diagnostics and Pharma with data insights from the clinical practice. For over 125 years, sustainability has been an integral part of Roche's business. As a science-driven company, our greatest contribution to society is developing innovative medicines and diagnostics that help people live healthier lives. Roche is committed to the Science Based Targets initiative and the Sustainable Markets Initiative to achieve net zero by 2045. Genentech, in the United States, is a wholly owned member of the Roche Group. Roche is the majority shareholder in Chugai Pharmaceutical, Japan. For more information, please visit * The pre-specified external control used contemporary datasets taken from three separate studies in Duchenne, two randomised controlled clinical trials and one natural history study, creating a prospectively defined consolidated comparison group of individuals with Duchenne, matched for variables, including age, steroid usage, baseline NSAA and timed function tests with the EMBARK part one treated patients. The prospectively defined propensity score analysis allows for a robust and rigorous balancing of multiple variables. All trademarks used or mentioned in this release are protected by Global Media RelationsPhone: +41 61 688 8888 / e-mail: Hans Trees, PhDPhone: +41 79 407 72 58 Sileia UrechPhone: +41 79 935 81 48 Nathalie AltermattPhone: +41 79 771 05 25 Lorena CorfasPhone: +41 79 568 24 95 Simon GoldsboroughPhone: +44 797 32 72 915 Karsten KleinePhone: +41 79 461 86 83 Nina MählitzPhone: +41 79 327 54 74 Kirti PandeyPhone: +49 172 6367262 Yvette PetillonPhone: +41 79 961 92 50 Dr Rebekka SchnellPhone: +41 79 205 27 03 Roche Investor Relations Dr Bruno EschliPhone: +41 61 68-75284e-mail: Dr Sabine BorngräberPhone: +41 61 68-88027e-mail: Dr Birgit MasjostPhone: +41 61 68-84814e-mail: Investor Relations North America Loren KalmPhone: +1 650 225 3217e-mail: Attachment 27012025_MR_ElevidysEMBARK_en
