Elevidys (delandistrogene moxeparvovec-rokl) / Sarepta Therap, Nationwide Children's, Roche - LARVOL DELTA

Conservative Management of Acute Myocarditis and Thrombotic Microangiopathy after Elevidys (Delandistrogene Moxeparvovec) (ASGCT 2025) - No abstract available

Late-breaking abstract • Cardiovascular • Inflammation

3-Year Functional Outcomes of Patients With Duchenne Muscular Dystrophy: Pooled Delandistrogene Moxeparvovec Clinical Trial Data vs External Controls (ASGCT 2025) - No abstract available

Clinical • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Long-Term Functional Outcomes and Safety Following Delandistrogene Moxeparvovec Treatment in DMD: EMBARK 2-Year Results (ASGCT 2025) - No abstract available

Clinical • Duchenne Muscular Dystrophy

Assessment of Cardiac Outcomes in Delandistrogene Moxeparvovec Clinical Trials for Duchenne Muscular Dystrophy (ASGCT 2025) - No abstract available

Clinical • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Sarepta’s Duchenne Gene Therapy Placed on Hold in Europe Pending Death Investigation (BioSpace) - "The European Medicines Agency has requested that Sarepta Therapeutics and partner Roche pause a trio of clinical trials of their Duchenne muscular dystrophy gene therapy Elevidys in response to a patient death that occurred outside the scope of the studies. In a letter addressed to the World Duchenne Organization and dated March 31, Roche shared that it was pausing Phase I, Phase II and Phase III trials all studying the gene therapy for treating Duchenne muscular dystrophy (DMD)."

Trial suspension • Duchenne Muscular Dystrophy

Why It Is Still Important to Ensure the Price is Right: Updates to Access Restrictions for Therapies Treating Duchenne Muscular Dystrophy (ISPOR 2025) - "Corticosteroids (Emflaza and Amagree) are excluded as they have significantly different value and price considerations. Coverage criteria aligned to trial criteria is to ensure appropriate use. Duvyzat's parity access to existing agents is likely a product of similar price-value alignment. If clinical benefit is unclear and price is high (e.g., non-ambulatory Elevidys), there is a prevailing risk of stakeholders adding restrictions (e.g., step edits) or only approving on a case-by-case basis (i.e., non-formulary)."

Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Roche to share latest scientific advancements from its neuromuscular portfolio at Muscular Dystrophy Association (MDA) 2025 conference (GlobeNewswire) - "Roche...announced today that it will present new data at the Muscular Dystrophy Association (MDA) conference, 16-19 March, 2025, in Dallas, Texas, from its neuromuscular portfolio, including 12 oral and poster presentations....Late-breaking oral on Elevidys’ Embark two-year data and pooled analysis of Study 101, 102 and Endeavor, demonstrated clinically meaningful and statistically significant improvements across key measures of motor function in boys with Duchenne muscular dystrophy (DMD)....At week 52, results showed stabilisation or slowing of disease progression in Elevidys-treated patients compared to placebo-treated patients. At week 104, MRI changes from baseline continued to generally favour Elevidys versus placebo at week 52 across muscles and muscle groups."

Clinical data • Duchenne Muscular Dystrophy

A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Imlifidase Infusion in Participants With Duchenne Muscular Dystrophy (DMD) Determined to Have Pre-existing Antibodies to Recombinant Adeno-Associated Virus Serotype (rAAVrh74) (clinicaltrials.gov) - P1 | N=6 | Enrolling by invitation | Sponsor: Sarepta Therapeutics, Inc. | Trial completion date: Sep 2026 ➔ Oct 2027 | Trial primary completion date: Jan 2025 ➔ Jan 2026

Trial completion date • Trial primary completion date • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy

ENVOL: A Gene Delivery Study to Evaluate the Safety and Expression of Delandistrogene Moxeparvovec in Participants Under the Age of Four With Duchenne Muscular Dystrophy (DMD) (clinicaltrials.gov) - P2 | N=21 | Recruiting | Sponsor: Hoffmann-La Roche | Trial primary completion date: Nov 2032 ➔ May 2033

Trial primary completion date • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy • TRA

The latest developments in synthetic approaches to duchenne muscular dystrophy. (PubMed, Expert Rev Neurother) - "Although corticosteroids remain the standard treatment, newly approved drugs such as exon-skipping therapies, vamorolone, delandistrogene moxeparvovec, and givinostat provide new treatment options...The accelerated FDA review process has enabled faster approval of new medications; however many have provided minimal clinical benefit to patients. Despite these challenges, continued drug development and innovative research offer hope to patients."

Journal • Review • Cardiomyopathy • Cardiovascular • Duchenne Muscular Dystrophy • Fibrosis • Gene Therapies • Genetic Disorders • Immunology • Muscular Dystrophy • Respiratory Diseases

Roche announces new results from EMBARK demonstrating significant sustained benefits of Elevidys in ambulatory individuals with Duchenne muscular dystrophy (DMD) (Roche Press Release) - P3 | N=126 | EMBARK (NCT05096221) | Sponsor: Sarepta Therapeutics, Inc. | "Roche...announced today positive topline results from year two of the EMBARK trial....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....No new safety signals observed further reinforcing the consistent and manageable safety profile observed with Elevidys to date....Detailed results from year two of the EMBARK study will be shared at an upcoming medical meeting and discussed with health authorities."

P3 data: top line • Duchenne Muscular Dystrophy

Is duchenne gene therapy a suitable treatment despite its immunogenic class effect? (PubMed, Expert Opin Drug Saf) - "The FDA's recent approval of delandistrogene moxeparvovec (Elevidys) underscores the promise of gene replacement therapies for dystrophin restoration in DMD patients...CRISPR/Cas9 therapies, while promising, face significant regulatory and safety challenges that hinder their clinical application. Optimal DMD therapies should target both skeletal and cardiac muscles to be truly effective."

Journal • Review • Cardiomyopathy • Cardiovascular • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy

EMBARK: A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) (clinicaltrials.gov) - P3 | N=126 | Completed | Sponsor: Sarepta Therapeutics, Inc. | Active, not recruiting ➔ Completed

Trial completion • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy

ENVOL: A Gene Delivery Study to Evaluate the Safety and Expression of Delandistrogene Moxeparvovec in Participants Under the Age of Four With Duchenne Muscular Dystrophy (DMD) (clinicaltrials.gov) - P2 | N=21 | Recruiting | Sponsor: Hoffmann-La Roche | Trial completion date: Nov 2032 ➔ May 2033

Trial completion date • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Acute Liver Injury Following Delandistrogene Moxeparvovec Gene Therapy Requiring Intravenous Immunoglobulin. (PubMed, Pediatr Neurol) - No abstract available

Gene therapy • Journal • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Hepatology • Liver Failure • Muscular Dystrophy

Neuromuscular diseases: genomics-driven advances. (PubMed, Genomics Inform) - "In spinal muscular atrophy (SMA), therapies like nusinersen, onasemnogene abeparvovec, and risdiplam have dramatically improved patient outcomes. Similarly, Duchenne muscular dystrophy (DMD) has seen significant progress, most notably with the FDA approval of delandistrogene moxeparvovec, the first micro-dystrophin gene therapy. Despite these advancements, challenges remain, including the rarity of many NMDs, genetic heterogeneity, and the high costs associated with genomic technologies and therapies. Continued progress in gene therapy, RNA-based therapeutics, and personalized medicine holds promise for further breakthroughs in the management of these debilitating diseases."

Journal • Review • CNS Disorders • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Movement Disorders • Muscular Atrophy • Muscular Dystrophy • Rare Diseases

Long-Term Survival and Myocardial Function Following Systemic Delivery of Delandistrogene Moxeparvovec in DMDMDX Rats. (PubMed, Hum Gene Ther) - "Transgene expression was maintained up to >25 months and micro-dystrophin expression was broadly distributed across skeletal and cardiac muscle. Taken together, these findings demonstrate long-term cardiac efficacy and improved survival following delandistrogene moxeparvovec treatment in DMDMDX rats."

Journal • Preclinical • Cardiomyopathy • Cardiovascular • Duchenne Muscular Dystrophy • Fibrosis • Gene Therapies • Genetic Disorders • Heart Failure • Immunology • Muscular Dystrophy

Willingness to Pay for Ultra-Rare Diseases in US, Germany, France, UK, and Japan: A Comparative Study (ISPOR-EU 2024) - "Elevidys (DMD) with a WAC price of $3.2 million in US and Upstaza (AADC deficiency) with a list price of €3.5million in France are amongst the highest priced treatments globally... WIP for treatments for ultra rare diseases specifically gene therapies is very high and HTA institutions and payers have recognised the value of curative treatments reflected through positive HTA and price outcomes."

Gene Therapies • Genetic Disorders • Pediatrics • Rare Diseases

What Are the Key Themes and Sentiments Captured Through Social Listening in Response to Recent Changes in the Duchenne Muscular Dystrophy Treatment Landscape? (ISPOR-EU 2024) - " Using a social media listening platform (Brandwatch), we assessed the difference in stakeholder sentiment before and after the fordadistrogene movaparvovec announcement (June 12 th 2024), and after the announcement of the delandistrogene moxeparvovec-rokl approval (June 20 th 2024). The variability in sentiment in the DMD population over a relatively short time period confirms the ability of social media listening to capture patient perspectives quickly. However, this variability in sentiment over a 10 day time-period also points to a need for methodological scrutiny to reduce bias in social media listening data collection."

Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

Caregiver Global Impression Observations from EMBARK: A Phase 3 Study Evaluating Delandistrogene Moxeparvovec in Ambulatory Patients with Duchenne Muscular Dystrophy. (PubMed, Neurol Ther) - P3 | "These exploratory findings captured by caregiver-reported outcomes add to the totality of evidence that supports the clinical benefits of delandistrogene moxeparvovec for patients with DMD."

Clinical • Journal • P3 data • CNS Disorders • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy

Response to Hamid et al., "Equitable Access of Delandistrogene Moxeparvovec for Patients With Duchenne Muscular Dystrophy". (PubMed, Pediatr Neurol) - No abstract available

Journal • Duchenne Muscular Dystrophy • Genetic Disorders • Muscular Dystrophy

HORIZON: A Gene Transfer Therapy to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Therapeutic Plasma Exchange (Plasmapheresis) in Participants With Duchenne Muscular Dystrophy (DMD) and Pre-existing Antibodies to AAVrh74 (clinicaltrials.gov) - P1 | N=16 | Recruiting | Sponsor: Sarepta Therapeutics, Inc. | Not yet recruiting ➔ Recruiting

Enrollment open • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy

AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial. (PubMed, Nat Med) - P3 | "Safety was manageable and consistent with previous delandistrogene moxeparvovec trials. ClinicalTrials.gov: NCT05096221."

Gene therapy • Journal • P3 data • CNS Disorders • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy

HORIZON: A Gene Transfer Therapy to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Therapeutic Plasma Exchange (Plasmapheresis) in Participants With Duchenne Muscular Dystrophy (DMD) and Pre-existing Antibodies to AAVrh74 (clinicaltrials.gov) - P1 | N=16 | Not yet recruiting | Sponsor: Sarepta Therapeutics, Inc.

New P1 trial • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy

ENVISION: A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD) (clinicaltrials.gov) - P3 | N=148 | Recruiting | Sponsor: Sarepta Therapeutics, Inc. | Trial completion date: Jan 2027 ➔ Jun 2028 | Trial primary completion date: Jan 2026 ➔ May 2027

Trial completion date • Trial primary completion date • Duchenne Muscular Dystrophy • Gene Therapies • Genetic Disorders • Muscular Dystrophy