RegulationFast Track (United States), Accelerated Approval (United States), Orphan Drug (United States), Rare Pediatric Disease (United States), Orphan Drug (European Union), Orphan Drug (Japan), Paediatric investigation plan (European Union), Priority Review (United States)

Structure/Sequence

Sequence Code 453698636

Clinical Trials associated with Delandistrogene moxeparvovec

NCT06597656

/ RecruitingPhase 1

An Open-Label, Systemic Gene Delivery Study to Evaluate the Safety, Tolerability and Expression of Delandistrogene Moxeparvovec Following Plasmapheresis in Subjects With Duchenne Muscular Dystrophy and Pre-existing Antibodies to AAVrh74

This is a gene transfer therapy study evaluating the safety of and delandistrogene moxeparvovec dystrophin protein expression from delandistrogene moxeparvovec following therapeutic plasma exchange (plasmapheresis) in ambulatory male participants with DMD and pre-existing antibodies to AAVrh74 over a period of 58 weeks.

Start Date18 Sep 2024

Sponsor / Collaborator

Sarepta Therapeutics, Inc.

NCT06270719

/ RecruitingNot Applicable

A Long-term Multicenter Prospective Observational Study Evaluating the Comparative Effectiveness and Safety of Sarepta Gene Transfer Therapy vs. Standard of Care in Participants With Duchenne Muscular Dystrophy Under Conditions of Routine Clinical Practice

This is a multicenter, prospective, observational Phase 4 study in the United States. The study is designed to collect both medical history and prospective data on Duchenne muscular dystrophy (DMD) treatment outcomes in participants receiving delandistrogene moxeparvovec as part of clinical care, compared to participants with DMD receiving or prescribed to start chronic glucocorticoid treatment at baseline in routine clinical practice.

Start Date07 Feb 2024

Sponsor / Collaborator

Sarepta Therapeutics, Inc.

NCT06241950

/ Enrolling by invitationPhase 1

An Open-Label, Systemic Gene Delivery Study to Evaluate the Safety, Tolerability and Expression of SRP-9001 in Association With Imlifidase in Subjects With Duchenne Muscular Dystrophy With Pre-existing Antibodies to rAAVrh74

This is a gene transfer therapy study evaluating the safety of delandistrogene moxeparvovec and delandistrogene moxeparvovec dystrophin expression in association with imlifidase, in participants with DMD with pre-existing antibodies to rAAVrh74 over a period of 104 weeks.

Start Date29 Jan 2024

Sponsor / Collaborator

Sarepta Therapeutics, Inc.

[+1]

100 Clinical Results associated with Delandistrogene moxeparvovec

100 Translational Medicine associated with Delandistrogene moxeparvovec

100 Patents (Medical) associated with Delandistrogene moxeparvovec

Literatures (Medical) associated with Delandistrogene moxeparvovec

10 Jun 2025·NEUROLOGY

Delandistrogene Moxeparvovec Gene Therapy in Individuals With Duchenne Muscular Dystrophy: Evidence in Focus

Review

Author: Dowling, James J. ; Rose, Sean C. ; Parsons, Julie A. ; Butterfield, Russell J. ; Puskala Hamel, Katie ; Silsbee, Heather M. ; Caller, Tracie Anne ; Servais, Laurent ; Bharadwaj, Jyoti ; Tolchin, Benjamin ; Oskoui, Maryam

This Evidence in Focus reviews the current evidence on the efficacy and adverse effects of delandistrogene moxeparvovec in patients with Duchenne muscular dystrophy (DMD) and presents clinical considerations regarding use. The author panel systematically reviewed available clinical trial data on delandistrogene moxeparvovec in patients with DMD. The risk of bias was evaluated using the American Academy of Neurology's 2017 therapeutic classification of evidence scheme. Safety information, regulatory decisions, and clinical context were also reviewed. Six clinical trials were identified, of which 4 had peer-reviewed data available. From the 4 studies with available data (2 Class I and 2 Class III), exposure data are available on 134 boys, of which 128 are ambulatory and aged ≥4 to <8 years. Both Class I studies failed to meet the primary functional motor outcome as assessed by change in the North Star Ambulatory Assessment score. Several secondary functional motor outcomes demonstrated improvement in the treatment group with small effect sizes, not meeting statistical significance from hierarchical analysis. Corticosteroid dose exposure was higher in the treatment group in the first 12 weeks after infusion, potentially contributing to measured differences between groups. Safety outcomes were similar across studies with multiple treatment-related adverse events, including peri-infusion effects, immune myositis and myocarditis, thrombocytopenia, and liver toxicity. One death has been reported in an individual who was treated with delandistrogene moxeparvovec outside of a trial. Despite not demonstrating efficacy in its primary outcome, delandistrogene moxeparvovec has been approved by the US Food and Drug Administration (FDA) for use in boys with DMD. This decision was supported by the relative safety of the product and secondary outcome measures data in the phase 3 clinical trial. As the drug may now be actively prescribed in the United States and other countries after FDA approval, providers should be aware of the limitations of the treatment and the need to monitor for immune-related side effects including myocarditis, liver injury, and thrombocytopenia, which may require expanded clinical infrastructure. Additional clinical trials and careful collection of real-world evidence from treated patients will be essential to establish short-term and long-term effectiveness and inform understanding of benefits and risks of delandistrogene moxeparvovec across the lifespan.

04 Mar 2025·Expert Review of Neurotherapeutics

The latest developments in synthetic approaches to Duchenne muscular dystrophy

Review

Author: Johnson, Lucy M. ; Pulskamp, Tariq G. ; Berlau, Daniel J.

INTRODUCTION:

Duchenne muscular dystrophy (DMD) is a rare X-linked genetic disorder caused by mutations in the dystrophin gene, leading to an almost complete absence of dystrophin, which is essential for muscle cell structure and function. This resulting muscle deterioration and fibrosis, eventually causes respiratory failure and cardiomyopathy. While there is currently no cure, existing therapies aim to prolong survival and alleviate symptoms.

AREAS COVERED:

This paper reviews current and emerging therapies for DMD, focusing on their safety and efficacy. Although corticosteroids remain the standard treatment, newly approved drugs such as exon-skipping therapies, vamorolone, delandistrogene moxeparvovec, and givinostat provide new treatment options. Additionally, future therapies, including gene therapy, stem cell treatments, and anti-fibrotic agents, show promise for clinical application.

EXPERT OPINION:

Advancements in DMD treatments have expanded patient options. While gene therapy offers potential for correcting the genetic defect and alleviating symptoms, corticosteroids remain the most cost-effective and well-researched treatment. This is partly due to the lack of compelling long-term safety and efficacy data for gene therapies. 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.

01 Mar 2025·MOLECULAR THERAPY

A comprehensive atlas of AAV tropism in the mouse

Article

Author: Cox, Aaron R ; Dickinson, Mary E ; Lutz, Cathleen M ; Lagor, William R ; Gaitan, Yaned ; Bulcha, Jote ; Hartig, Sean M ; Chuecos, Marcel A ; Suarez, Carlos Flores ; Gao, Guangping ; Hsu, Chih-Wei ; Wang, Dan ; Snow, Kathy J ; Duryea, Jeff ; Walkey, Christopher J ; Martinez, Alexa E ; Hilsenbeck, Susan G ; Hannigan, Seth ; Mirochnitchenko, Oleg ; De Giorgi, Marco ; Alves-Bezerra, Michele ; Ljungberg, M Cecilia ; Saville, Ethan ; Lanza, Denise G ; Murray, Stephen A ; Heaney, Jason D

Gene therapy with adeno-associated virus (AAV) vectors requires knowledge of their tropism within the body. Here we analyze the tropism of 10 naturally occurring AAV serotypes (AAV3B, AAV4, AAV5, AAV6, AAV7, AAV8, AAV9, AAVrh8, AAVrh10, and AAVrh74) following systemic delivery into male and female mice. A transgene-expressing ZsGreen and Cre recombinase was used to identify transduction in a cell-dependent manner based on fluorescence. Cre-driven activation of tdTomato fluorescence offered superior sensitivity for transduced cells. All serotypes except AAV3B and AAV4 had high liver tropism. Fluorescence activation revealed transduction of unexpected tissues, including adrenals, testes, and ovaries. Rare transduced cells within tissues were also readily visualized. Biodistribution of AAV genomes correlated with fluorescence, except in immune tissues. AAV4 was found to have a pan-endothelial tropism while also targeting pancreatic beta cells. This public resource enables selection of the best AAV serotypes for basic science and preclinical applications in mice.

254

News (Medical) associated with Delandistrogene moxeparvovec

21 May 2025

·investorrelations.sarepta.com

Sarepta Provides Update on UK Dosing in ENVISION Study of ELEVIDYS for the treatment of Duchenne Muscular Dystrophy

CAMBRIDGE, Mass.--(BUSINESS WIRE)--May 21, 2025-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, shared the following update related to ELEVIDYS (delandistrogene moxeparvovec-rokl), the only approved gene therapy for patients with Duchenne muscular dystrophy. We have received feedback from the Medicines & Healthcare products Regulatory Agency (MHRA) in the United Kingdom (U.K.) that dosing may continue uninterrupted in ENVISION, study SRP-9001-303. ENVISION is a global, randomized, double-blind, placebo-controlled Phase 3 study of ELEVIDYS in non-ambulatory and older ambulatory individuals with Duchenne. About ELEVIDYS (delandistrogene moxeparvovec-rokl) ELEVIDYS (delandistrogene moxeparvovec-rokl) is a single-dose, adeno-associated virus (AAV)-based gene transfer therapy for intravenous infusion designed to address the underlying genetic cause of Duchenne muscular dystrophy – mutations or changes in the DMD gene that result in the lack of dystrophin protein – through the delivery of a transgene that codes for the targeted production of ELEVIDYS micro-dystrophin in skeletal muscle. In the U.S., ELEVIDYS is indicated for the treatment of Duchenne muscular dystrophy (DMD) in individuals at least 4 years of age. For patients who are ambulatory and have a confirmed mutation in the DMD gene For patients who are non-ambulatory and have a confirmed mutation in the DMD gene. The DMD indication in non-ambulatory patients is approved under accelerated approval based on expression of ELEVIDYS micro-dystrophin in skeletal muscle. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). IMPORTANT SAFETY INFORMATION CONTRAINDICATION: ELEVIDYS is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the DMD gene. WARNINGS AND PRECAUTIONS: Infusion-related Reactions: Infusion-related reactions, including hypersensitivity reactions and anaphylaxis, have occurred during or up to several hours following ELEVIDYS administration. Closely monitor patients during administration and for at least 3 hours after the end of infusion. If symptoms of infusion-related reactions occur, slow, or stop the infusion and give appropriate treatment. Once symptoms resolve, the infusion may be restarted at a lower rate. ELEVIDYS should be administered in a setting where treatment for infusion-related reactions is immediately available. Discontinue infusion for anaphylaxis. Acute Serious Liver Injury: Acute serious liver injury has been observed with ELEVIDYS, and administration may result in elevations of liver enzymes (such as GGT, GLDH, ALT, AST) or total bilirubin, typically seen within 8 weeks. Patients with preexisting liver impairment, chronic hepatic condition, or acute liver disease (e.g., acute hepatic viral infection) may be at higher risk of acute serious liver injury. Postpone ELEVIDYS administration in patients with acute liver disease until resolved or controlled. Prior to ELEVIDYS administration, perform liver enzyme test and monitor liver function (clinical exam, GGT, and total bilirubin) weekly for the first 3 months following ELEVIDYS infusion. Continue monitoring if clinically indicated, until results are unremarkable (normal clinical exam, GGT, and total bilirubin levels return to near baseline levels). Systemic corticosteroid treatment is recommended for patients before and after ELEVIDYS infusion. Adjust corticosteroid regimen when indicated. If acute serious liver injury is suspected, consultation with a specialist is recommended. Immune-mediated Myositis: In clinical trials, immune-mediated myositis has been observed approximately 1 month following ELEVIDYS infusion in patients with deletion mutations involving exon 8 and/or exon 9 in the DMD gene. Symptoms of severe muscle weakness, including dysphagia, dyspnea, and hypophonia, were observed. Limited data are available for ELEVIDYS treatment in patients with mutations in the DMD gene in exons 1 to 17 and/or exons 59 to 71. Patients with deletions in these regions may be at risk for a severe immune-mediated myositis reaction. Advise patients to contact a physician immediately if they experience any unexplained increased muscle pain, tenderness, or weakness, including dysphagia, dyspnea, or hypophonia, as these may be symptoms of myositis. Consider additional immunomodulatory treatment (immunosuppressants [e.g., calcineurin-inhibitor] in addition to corticosteroids) based on patient’s clinical presentation and medical history if these symptoms occur. Myocarditis: Acute serious myocarditis and troponin-I elevations have been observed following ELEVIDYS infusion in clinical trials. If a patient experiences myocarditis, those with pre-existing left ventricle ejection fraction (LVEF) impairment may be at higher risk of adverse outcomes. Monitor troponin-I before ELEVIDYS infusion and weekly for the first month following infusion and continue monitoring if clinically indicated. More frequent monitoring may be warranted in the presence of cardiac symptoms, such as chest pain or shortness of breath. Advise patients to contact a physician immediately if they experience cardiac symptoms. Preexisting Immunity against AAVrh74: In AAV-vector based gene therapies, preexisting anti-AAV antibodies may impede transgene expression at desired therapeutic levels. Following treatment with ELEVIDYS, all patients developed anti-AAVrh74 antibodies. Perform baseline testing for presence of anti-AAVrh74 total binding antibodies prior to ELEVIDYS administration. ELEVIDYS administration is not recommended in patients with elevated anti-AAVrh74 total binding antibody titers greater than or equal to 1:400. Adverse Reactions: The most common adverse reactions (incidence ≥5%) reported in clinical studies were vomiting, nausea, liver injury, pyrexia, and thrombocytopenia. Report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088. You may also report side effects to Sarepta Therapeutics at 1-888-SAREPTA (1-888-727-3782). For further information, please see the full Prescribing Information. About Sarepta Therapeutics Sarepta is on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold leadership positions in Duchenne muscular dystrophy (Duchenne) and limb-girdle muscular dystrophies (LGMDs) and are building a robust portfolio of programs across muscle, central nervous system, and cardiac diseases. For more information, please visit www.sarepta.com or follow us on LinkedIn, X, Instagram and Facebook. Internet Posting of Information We routinely post information that may be important to investors in the 'For Investors' section of our website at www.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us. Forward-Looking Statements This statement contains “forward-looking statements.” Any statements that are not statements of historical fact may be deemed to be forward-looking statements. Words such as “believe,” “anticipate,” “plan,” “expect,” “will,” “may,” “intend,” “prepare,” “look,” “potential,” “possible” and similar expressions are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements relating to our future operations, research and development programs, and clinical trials. Actual results could materially differ from those stated or implied by these forward-looking statements as a result of such risks and uncertainties. Known risk factors include the following: our products or product candidates may be perceived as insufficiently effective, unsafe or may result in unforeseen adverse events; our products or product candidates may cause undesirable side effects that result in significant negative consequences following any marketing approval; and those risks identified under the heading “Risk Factors” in our most recent Annual Report on Form 10-K for the year ended December 31, 2024 filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company, which you are encouraged to review. Any of the foregoing risks could materially and adversely affect the Company’s business, results of operations and the trading price of Sarepta’s common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained herein. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof, except as required by law. View source version on businesswire.com: https://www.businesswire.com/news/home/20250521736806/en/ Investor Contact: Ian Estepan 617-274-4052 iestepan@sarepta.com Media Contacts: Tracy Sorrentino 617-301-8566 tsorrentino@sarepta.com Kara Hoeger 617-710-3898 KHoeger@sarepta.com Source: Sarepta Therapeutics, Inc.

Phase 3Gene TherapyAccelerated Approval

19 May 2025

·www.biospace.com

ASGCT Roundup: Sarepta, Rocket, Neurogene and the World’s First Tailored CRISPR Therapy

iStock, Anton Vierietin Taking center stage at the American Society of Gene and Cell Therapy meeting was the first-ever reported case of a personalized i n vivo CRISPR editing therapy, which substantially eased the symptom burden in an infant. Over the weekend, the American Society of Gene and Cell Therapy held its annual meeting in New Orleans, hosting some of the space’s most cutting-edge research and spotlighting clinical findings that could have far-reaching implications on medical practice. BioSpace highlights some notable presentations here. A World First: Personalized CRISPR Therapy Arguably the biggest news out of ASGCT was the infant that has been treated with a “customized” base-editing therapy, which greatly eased the burden of the infant’s ultra-rare disease. The case, also published in the New England Journal of Medicine , focused on a male infant who presented with lethargy and respiratory distress within 48 hours of birth. He would eventually be diagnosed with severe carbamoyl-phosphate synthetase 1 (CSP-1) deficiency , a rare disease involving the toxic build-up of nitrogen in the body. Approximately half of babies diagnosed with the condition die “in early infancy,” according to the paper. However, after two infusions of a customized CRISPR-based gene editing therapy at 7 and 8 months of age, the patient was able to gradually increase dietary protein intake, which would otherwise have to be strongly restricted in patients with CSP-1 deficiency. Median blood ammonia levels dropped and doctors were also able to scale back nitrogen-scavenging medication. There were no severe safety concerns, according to the researchers, though they did detect “transient” elevations in liver enzymes as well as two viral infections within 4 weeks after the second dose. Rocket Shares ‘Encouraging’ Data for Cardiomyopathy Gene Therapy On Thursday, Rocket Pharmaceuticals revealed that its investigational gene therapy RP-A601 normalized right ventricle systolic function in patients with plakophilin-2 related arrhythmogenic cardiomyopathy. RP-A601 also suppressed or completely stabilized off-beat contractions of the ventricles. Aside from these clinical improvements, patients treated with RP-A601 likewise showed significantly better quality of life and functional performance. In a note to investors on Friday, BMO Capital Markets analysts called these findings “encouraging,” adding that they suggest “strong gene expression” alongside improvements in functional endpoints. On safety, the analysts noted that one patient developed a serious adverse event—Rocket did not specify what this toxicity was, revealing only that it “resolved without clinical sequelae within two months post-treatment”—though this safety signal is likely to be linked to immunomodulator use, according to BMO. While the serious safety event “can raise investor questions,” BMO argued that “the overall benefit/risk pro favorable given lack of approved disease-modifying treatments” in this space. Sarepta Provides More Cardio Color for Elevidys One of the most talked-about names at the ASGCT meeting was Elevidys, Sarepta Therapeutics ’ gene therapy for Duchenne muscular dystrophy. After all, the biologic was rocked by a patient death in March—a development that caused the biotech’s shares to crater by 22%. Sarepta sought to shore up confidence in Elevidys at ASGCT, presenting consolidated cardiovascular outcomes from four studies totaling 218 patients. Results showed that cardiac troponin levels, a marker of heart damage, fluctuated following treatment, though these were largely asymptomatic. Cardiac MRI analyses also showed that Elevidys did not result in meaningful left ventricular changes. The study also found two cases of myocarditis that arose “within days” of Elevidys treatment, though these resolved within three weeks. Overall, results showed that Elevidys had a manageable cardiac safety pro five years of follow-up. Also at ASGCT, Sarepta provided Elevidys data in ambulatory children aged 8- to 9-years—information that the space has been eagerly awaiting . These latest data show that Elevidys can elicit “ statistically significant and clinically meaningful ” functional improvements in older kids, indicating that the gene therapy can stabilize disease or slow its progression, according to Sarepta. Neurogene Unveils New Safety Protocol for Rett Syndrome Gene Therapy After Patient Death In November 2024, Neurogene reported that a young patient had died in its Phase I/II study for NGN-401, an investigational gene therapy for Rett Syndrome. A few days earlier, the patient, who received the 3E15 vg dose of NGN-401, was revealed to be in critical condition after showing signs of systemic hyperinflammation. At ASGCT, Neurogene detailed its new safety monitoring and treatment system that would allow it to detect and reverse this hyperinflammatory syndrome. In particular, the biotech conducted daily surveillance in the first week after treating patients, looking out for what it called the “three Fs:” fever, falling blood counts and elevated ferritin levels. Neurogene also implemented standard algorithms to treat systemic hyperinflammation in its trial, including high-dose corticosteroid intervention, followed by an IL-1 receptor agonist. Neurogene is moving forward with the lower, 1E15 vg dose of NGN-401, which is in Phase I/II development. The biotech “is not aware of any case of” systemic hyperinflammation at this dose level, as per a news release on Friday.

Gene TherapyClinical ResultClinical Study

16 May 2025

·firstwordpharma.com

Rocket plans pivotal trial for cardiac gene therapy, but concerns linger on accelerated pathway

Safety and early functional results from three patients who received Rocket Pharmaceuticals' heart disease gene therapy were enough to convince the drugmaker to begin planning a pivotal study of RP-A601. But convincing the FDA's new head of the Center for Biologics Evaluation and Research (CBER) — who has criticised the accelerated approval pathway, particularly for gene therapies — may prove a bigger hurdle. Rocket's Thursday presentation at the American Society of Gene and Cell Therapy (ASGCT) meeting featured Phase I data from three patients with plakophilin-2 related arrhythmogenic cardiomyopathy (PKP2-ACM) who received a single 8x1013 GC/kg dose of RP-A601. The gene therapy comprises a recombinant adeno-associated serotype rh74 capsid containing a functional version of the human PKP2 transgene (AAVrh74.PKP2).Biomarker increases, manageable safetyNo dose-limiting toxicities were observed during the 12-month follow-up period. Two patients with low baseline PKP2 protein expression saw increases of 110% and 398%, respectively, at six months. All three patients showed early signs of improvements or stabilisation of their arrhythmia burden, heart function and quality of life. Specifically, they each had normal right ventricular systolic function at their latest follow-up appointment, and the rate of premature ventricular contractions fell between 9% to 63% from baseline at an evaluation occurring six to 12 months post-treatment. On the Kansas City Cardiomyopathy Questionnaire (KCCQ), which is used to evaluate quality of life, patients reported improvements between 34 to 41 points; a five-point or greater increase is considered clinically meaningful. According to the drugmaker, most treatment-related adverse events (AEs) were mild or moderate. One patient experienced severe AEs — Grade 3 transaminase elevation, Grade 4 pancreatitis, Grade 4 sepsis and Grade 3 bacteremia — but the symptoms, which Rocket believes to be associated with the immunomodulatory regimen, resolved within two months. The safety profile is "not entirely clean, but workable in a setting of high unmet need," Chardan analyst Geulah Livshits wrote in a note. While Rocket linked the SAEs to the immune regimen, its decision not to explore further dose escalation and to move directly into pivotal testing "makes sense," she said, "in light of fatal acute liver failure in a patient treated with Elevidys reported earlier this year, which has increased scrutiny on AAVrh.74 safety."The death of a 16-year-old boy who received Sarepta Therapeutics' Duchenne muscular dystrophy (DMD) gene therapy has slowed down US sales of the product and triggered holds on several European studies evaluating Elevidys (delandistrogene moxeparvovec-rokl) — and has sent the biotech's stock into a tailspin (see – Vital Signs: Is Sarepta now priced for a buyout?).Sarepta's problems potentially worsened this month after FDA Commissioner Marty Makary tapped Vinay Prasad to lead CBER (see – Vital Signs: What a new CBER director means for biotech and the accelerated approval pathway). Prasad has been a vocal critic of Elevidys' accelerated approval in 2023, and its subsequent label expansion last year into ambulatory and non-ambulatory boys aged four years and older. Sarepta announced plans on Friday to seek another supplementary approval for Elevidys in DMD patients as young as two years old. Rocket's use of the same AAV vector as Elevidys, and its accelerated approval hopes, may place it in Prasad's crosshairs.As BMO Capital Markets analyst Kostas Biliouris pointed out in a note, "[the accelerated approval] AA pathway availability is key" for Rocket. He remained optimistic that the drugmaker will be able to reach an accelerated agreement for RP-A601, though he noted the firm is now awaiting "feedback from the new CBER director."

Clinical ResultGene TherapyPhase 1

100 Deals associated with Delandistrogene moxeparvovec

External Link

KEGG	Wiki	ATC	Drug Bank
-	Delandistrogene moxeparvovec	-	-

R&D Status

10 top approved records.

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Indication	Country/Location	Organization	Date
Muscular Dystrophy, Duchenne	United States	Sarepta Therapeutics, Inc.	22 Jun 2023

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT05096221 (EMBARK, Company_Website) Manual	Phase 3	Muscular Dystrophy, Duchenne	14	ELEVIDYS	jibdtedfvx(qeclhubbrn) = lkpepwpwhj lklumarmmf (ubaebcvzrc ) View more	Positive	16 May 2025
				external control cohort	-
NCT04626674 (SRP-9001-103 \| ENDEAVOR, Company_Website) Manual	Phase 1	Muscular Dystrophy, Duchenne	13	ELEVIDYS (cohort 6, 2 years old at time of treatment)	ifljxukluv(sexlgsmejy) = gnmdvdxsbj xqmewfpyli (lelgnnxenq ) View more	Positive	16 May 2025
				ELEVIDYS (cohort 4, 3 years old at time of treatment)	ifljxukluv(sexlgsmejy) = bpmkuouoht xqmewfpyli (lelgnnxenq )
Delandistrogene Moxeparvovec Clinical Trials (ASGCT2025)	Not Applicable	Muscular Dystrophy, Duchenne Troponin-I (TnI)	156	Delandistrogene Moxeparvovec	uscdtzlaxk(ndigbrukse) = At week 52, cardiac MRI performed in a subset of patients from EMBARK revealed no relevant differences in cardiac function (including LVEF), volume, or mass between patients treated with delandistrogene moxeparvovec or placebo hgqyftfogo (nxucgswsei ) View more	Positive	13 May 2025
NCT05096221 (EMBARK \| SRP-9001-301, PipelineReview) Manual	Phase 3	Muscular Dystrophy, Duchenne	125	ELEVIDYS+Placebo (Crossover-Treated Patients)	fwyvplmynt(ioyxvoqsfa) = entexgxjts dfmctsjfgl (bszzoijmvm )	Positive	27 Jan 2025
NCT05096221 (EMBARK, Literature) Manual	Phase 3	Muscular Dystrophy, Duchenne	125	delandistrogene moxeparvovec	npiribsyhp(qbezykrusb) = pyoaznelxe ocxoouqcgh (cuxnzgaadv ) View more	Negative	09 Oct 2024
				Placebo	npiribsyhp(qbezykrusb) = wjqqkjbwqs ocxoouqcgh (cuxnzgaadv ) View more
NCT05096221 (EMBARK, ASGCT2024)	Phase 3	Muscular Dystrophy, Duchenne	125	Delandistrogene moxeparvovec	fumxksjiau(kzhpafpazv): P-Value = 0.24 View more	Positive	07 May 2024
				Delandistrogene Moxeparvovec (Placebo)
NCT04626674 (ENDEAVOR, Pubmed \| Ann Neurol) Manual	Phase 1	Muscular Dystrophy, Duchenne	20	Delandistrogene moxeparvovec	dwuayaxznn(mviwzcdzmx) = pmkdgsgaue wihjhpuoet (taapzlhgsw, 42.6) View more	Positive	01 Nov 2023
NCT05096221 (SRP-9001-301 \| EMBARK, Corporate Publications) Manual	Phase 3	Muscular Dystrophy, Duchenne	125	ELEVIDYS	vnsyrxrfqi(kfzrzccokp) = rwqiixebth reuiglwvbf (qlkecyzgqd ) Not Met	Positive	30 Oct 2023
				placebo	vnsyrxrfqi(kfzrzccokp) = qmsrodzhgk reuiglwvbf (qlkecyzgqd ) Not Met
FDA Manual	Not Applicable	Muscular Dystrophy, Duchenne	41	ELEVIDYS	dlflbedqrg(ctpfcboetq) = Most common adverse reactions across studies (incidence ≥5%) were vomiting and nausea, liver function test increased, pyrexia, and thrombocytopenia. xavddovmob (fjaxbismhb ) View more	Positive	22 Jun 2023
				Placebo
AAN2023	Not Applicable	Muscular Dystrophy, Duchenne	52	Delandistrogene moxeparvovec	fpemfytorc(ncisvssqkl) = dfskjgtfcp ahgyhfwoxf (umrqybcnpn )	-	25 Apr 2023

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