delandistrogene moxeparvovec

also known as Delandistrogene moxeparvovec component of elevidys · ELEVIDYS · SRP-9001

Gene therapy (AAV / viral vector) targets DMD

Developed for: Duchenne muscular dystrophy
Investigated by: Sarepta Therapeutics, Inc. · Hoffmann-La Roche · Hansa Biopharma AB

Regulatory milestones approvals, filings & regulatory actions · 1 recorded

Milestone	Jurisdiction	Brand	Indication	Date	Sentence it was read from
Refused (EMA)	EU (EMA)	Elevidys	—	2025-09-24	europa.eu ↗

Trials 10 · a red edge is where a trial was stopped

today NCT07542314 · Ph 4 NCT06597656 · Ph 1 NCT06241950 · Ph 1 NCT06128564 · Ph 2 NCT05967351 · Ph 3 NCT05881408 · Ph 3 NCT05096221 · Ph 3 NCT04626674 · Ph 1 NCT03769116 · Ph 1/2 NCT03375164 · Ph 1/2

20182019202020212022202320242025202620272028202920302031203220332034

Phase	Registry id	Dates	Indication	Sponsor	Status	Outcome
Phase 13 trials
Phase 1	NCT06597656	Sep 2024 → Aug 2025	Duchenne muscular dystrophy	Sarepta Therapeutics, Inc.	Terminated	No outcome recorded
Phase 1	NCT06241950	Jan 2024 → Oct 2025	Duchenne muscular dystrophy	Sarepta Therapeutics, Inc.	Terminated	No outcome recorded
Phase 1	NCT04626674	Nov 2020 → Dec 2027 expected	Duchenne muscular dystrophy	Sarepta Therapeutics, Inc.	Recruiting	No outcome recorded
Phase 1/22 trials
Phase 1/2	NCT03769116	Dec 2018 → Dec 2020	Duchenne muscular dystrophy	Sarepta Therapeutics, Inc.	Completed	No outcome recorded
Phase 1/2	NCT03375164	Jan 2018 → Apr 2023	Duchenne muscular dystrophy	Sarepta Therapeutics, Inc.	Completed	No outcome recorded
Phase 21 trial
Phase 2	NCT06128564	Nov 2023 → Mar 2026 overdue	Duchenne muscular dystrophy	Hoffmann-La Roche	Active not recruiting	No outcome recorded
Phase 33 trials
Phase 3	NCT05967351	Sep 2023 → Oct 2033 expected	Duchenne muscular dystrophy	Sarepta Therapeutics, Inc.	Enrolling by invitation	No outcome recorded
Phase 3	NCT05881408	May 2023 → May 2027 expected	Duchenne muscular dystrophy	Sarepta Therapeutics, Inc.	Active not recruiting	No outcome recorded
Phase 3	NCT05096221	Oct 2021 → Oct 2023	Duchenne muscular dystrophy	Sarepta Therapeutics, Inc.	Completed	No outcome recorded
Phase 41 trial
Phase 4	NCT07542314	Jul 2026 → Mar 2027 expected	Duchenne muscular dystrophy	Sarepta Therapeutics, Inc.	Not yet recruiting	No outcome recorded

Press releases naming this drug 46 releases

Date	Issuer	Release
2026-03-16	Sarepta Therapeutics, Inc.	Sarepta Announces that Screening and Enrollment are Underway in ENDEAVOR Cohort 8 to Evaluate Enhanced Immunosuppression Regimen as Part of ELEVIDYS Gene Therapy for Non-Ambulant Individuals with Duchenne sarepta.com ↗
2026-02-24	Sarepta Therapeutics, Inc.	Sarepta Therapeutics Announces Commercial Launch of ELEVIDYS in Japan sarepta.com ↗
2026-01-26	Sarepta Therapeutics, Inc.	Sarepta Announces Positive Topline Three-Year EMBARK Results Showing ELEVIDYS Significantly Slows Disease Progression on Key Functional Measures in Ambulatory Duchenne Patients sarepta.com ↗
2026-01-23	Sarepta Therapeutics, Inc.	Sarepta Therapeutics to Report 3-Year Topline Data from EMBARK Study of ELEVIDYS Gene Therapy in Ambulatory Individuals with Duchenne Muscular Dystrophy sarepta.com ↗
2025-11-25	Sarepta Therapeutics, Inc.	Sarepta Announces Approval to Begin ENDEAVOR Cohort 8 to Evaluate Enhanced Immunosuppression Regimen as Part of ELEVIDYS Gene Therapy for Non-Ambulant Individuals with Duchenne sarepta.com ↗
2025-11-14	Sarepta Therapeutics, Inc.	Sarepta Announces FDA’s Approval of Updated ELEVIDYS Prescribing Information sarepta.com ↗
2025-07-28	Sarepta Therapeutics, Inc.	FDA Informs Sarepta That It Recommends That Sarepta Remove Its Pause and Resume Shipments of ELEVIDYS for Ambulatory Individuals With Duchenne Muscular Dystrophy sarepta.com ↗
2025-07-25	Roche Global Development	Roche provides regulatory update on Elevidys™ gene therapy for Duchenne muscular dystrophy in the EU roche.com ↗
2025-07-25	Sarepta Therapeutics, Inc.	Sarepta Therapeutics Provides Clarifying Statement on ELEVIDYS sarepta.com ↗
2025-07-25	Sarepta Therapeutics, Inc.	Sarepta Therapeutics Acknowledges CHMP Negative Opinion for ELEVIDYS in the European Union sarepta.com ↗
2025-07-21	Sarepta Therapeutics, Inc.	Sarepta Therapeutics Announces Voluntary Pause of ELEVIDYS Shipments in the U.S. sarepta.com ↗
2025-07-18	Sarepta Therapeutics, Inc.	Sarepta Therapeutics Provides Statement on ELEVIDYS sarepta.com ↗
2025-07-16	Sarepta Therapeutics, Inc.	Sarepta Therapeutics Announces Strategic Restructuring and Pipeline Prioritization Plan to Maintain Long-term, Sustainable Growth and Provides Update on ELEVIDYS Label sarepta.com ↗
2025-06-15	Roche Global Development	[Ad hoc announcement pursuant to Art. 53 LR] Roche provides safety update on Elevidys™ gene therapy for Duchenne muscular dystrophy in non-ambulatory patients roche.com ↗
2025-06-15	Sarepta Therapeutics, Inc.	Sarepta Provides Safety Update for ELEVIDYS and Initiates Steps to Strengthen Safety in Non-Ambulatory Individuals with Duchenne sarepta.com ↗
2025-05-21	Sarepta Therapeutics, Inc.	Sarepta Provides Update on UK Dosing in ENVISION Study of ELEVIDYS for the treatment of Duchenne Muscular Dystrophy sarepta.com ↗
2025-05-13	Sarepta Therapeutics, Inc.	Sarepta Therapeutics Announces Approval in Japan of ELEVIDYS, a Gene Therapy to Treat Duchenne Muscular Dystrophy sarepta.com ↗
2025-04-04	Sarepta Therapeutics, Inc.	Sarepta Therapeutics Provides Update on ELEVIDYS sarepta.com ↗
2025-01-27	Roche Global Development	Roche announces new results from EMBARK demonstrating significant sustained benefits of Elevidys in ambulatory individuals with Duchenne muscular dystrophy (DMD) roche.com ↗
2025-01-27	Sarepta Therapeutics, Inc.	Sarepta Therapeutics Announces Results from Part 2 of the EMBARK Study Demonstrating Sustained Benefits and Disease Stabilization in Ambulatory Individuals with Duchenne Muscular Dystrophy Following Treatment with ELEVIDYS sarepta.com ↗
2024-06-20	Sarepta Therapeutics, Inc.	Sarepta Therapeutics Announces Expanded US FDA Approval of ELEVIDYS to Duchenne Muscular Dystrophy Patients Ages 4 and Above sarepta.com ↗
2024-02-16	Sarepta Therapeutics, Inc.	Sarepta Therapeutics Announces U.S. FDA Acceptance of an Efficacy Supplement to Expand the ELEVIDYS Indication sarepta.com ↗
2023-12-22	Sarepta Therapeutics, Inc.	Sarepta Therapeutics Submits Efficacy Supplement to Expand the ELEVIDYS Label to include Duchenne Muscular Dystrophy Patients without Restriction to Age or Ambulatory Status sarepta.com ↗
2023-10-30	Sarepta Therapeutics, Inc.	Sarepta Therapeutics Announces Topline Results from EMBARK, a Global Pivotal Study of ELEVIDYS Gene Therapy for Duchenne Muscular Dystrophy sarepta.com ↗
2023-06-22	Sarepta Therapeutics, Inc.	Sarepta Therapeutics Announces FDA Approval of ELEVIDYS, the First Gene Therapy to Treat Duchenne Muscular Dystrophy sarepta.com ↗
2023-05-24	Sarepta Therapeutics, Inc.	Sarepta Therapeutics Announces Update on Regulatory Review of SRP-9001 sarepta.com ↗
2023-05-12	Sarepta Therapeutics, Inc.	Sarepta Therapeutics Announces Positive Vote from U.S. FDA Advisory Committee Meeting for SRP-9001 Gene Therapy to Treat Duchenne Muscular Dystrophy sarepta.com ↗
2023-04-10	Sarepta Therapeutics, Inc.	Sarepta Therapeutics Announces that the Date of the Advisory Committee Meeting for SRP-9001 will be May 12, 2023 sarepta.com ↗
2023-03-16	Sarepta Therapeutics, Inc.	Sarepta Therapeutics Announces Advisory Committee Meeting will be Held for SRP-9001 sarepta.com ↗
2022-11-28	Sarepta Therapeutics, Inc.	Sarepta Therapeutics Announces That U.S. FDA has Accepted for Filing and Granted Priority Review for the Biologics License Application for SRP-9001, Sarepta’s Gene Therapy for the Treatment of Ambulant Individuals with Duchenne Muscular Dystrophy sarepta.com ↗
2022-09-29	Sarepta Therapeutics, Inc.	Sarepta Therapeutics Submits Biologics License Application for SRP-9001 for the Treatment of Ambulant Patients with Duchenne Muscular Dystrophy sarepta.com ↗
2022-07-29	Sarepta Therapeutics, Inc.	Sarepta Therapeutics Announces Intent to Submit an Accelerated Approval Biologics License Application for its Gene Therapy SRP-9001 to Treat Duchenne Muscular Dystrophy sarepta.com ↗
2022-07-06	Sarepta Therapeutics, Inc.	Sarepta Therapeutics’ Investigational Gene Therapy SRP-9001 for Duchenne Muscular Dystrophy Demonstrates Significant Functional Improvements Across Multiple Studies sarepta.com ↗
2022-07-05	Sarepta Therapeutics, Inc.	Sarepta Therapeutics to Share New Clinical Data and Integrated Analysis for SRP-9001, its Investigational Gene Therapy for the Treatment of Duchenne Muscular Dystrophy on July 6, 2022 at 8:30am ET sarepta.com ↗
2022-01-10	Sarepta Therapeutics, Inc.	Sarepta Therapeutics’ Gene Therapy SRP-9001 Shows Statistically Significant Functional Improvements Compared to Pre-specified Matched External Control in Part 2 of Study SRP-9001-102 for the Treatment of Duchenne Muscular Dystrophy sarepta.com ↗
2021-10-11	Sarepta Therapeutics, Inc.	Sarepta Therapeutics’ SRP-9001 Shows Sustained Functional Improvements in Multiple Studies of Patients with Duchenne sarepta.com ↗
2021-10-04	Sarepta Therapeutics, Inc.	Sarepta Therapeutics Announces Initiation of EMBARK, a Global Pivotal Study of SRP-9001, a Gene Therapy for the Treatment of Duchenne Muscular Dystrophy sarepta.com ↗
2021-05-18	Sarepta Therapeutics, Inc.	Sarepta Therapeutics’ Investigational Gene Therapy for the Treatment of Duchenne Muscular Dystrophy, SRP-9001, Demonstrates Robust Expression and Consistent Safety Profile Using Sarepta’s Commercial Process Material sarepta.com ↗
2021-05-17	Sarepta Therapeutics, Inc.	Sarepta Therapeutics to Share Expression and Safety Results from Study 103 (ENDEAVOR) Evaluating SRP-9001, its Investigational Gene Therapy for the Treatment of Duchenne Muscular Dystrophy sarepta.com ↗
2021-01-07	Sarepta Therapeutics, Inc.	Sarepta Therapeutics Announces Top-line Results for Part 1 of Study 102 Evaluating SRP-9001, its Investigational Gene Therapy for the Treatment of Duchenne Muscular Dystrophy sarepta.com ↗
2020-11-05	Sarepta Therapeutics, Inc.	Sarepta Therapeutics to Commence Dosing of the Next Study with Commercial Process Material for the SRP-9001 Gene Therapy Program for the Treatment of Duchenne Muscular Dystrophy sarepta.com ↗
2020-09-28	Sarepta Therapeutics, Inc.	Sarepta Therapeutics Reports Sustained Functional Improvement Two Years After Treatment with SRP-9001, its Investigational Micro-dystrophin Gene Therapy for Duchenne Muscular Dystrophy sarepta.com ↗
2020-09-09	Sarepta Therapeutics, Inc.	Sarepta Therapeutics Provides Program Update for SRP-9001, its Investigational Gene Therapy for the Treatment of Duchenne Muscular Dystrophy sarepta.com ↗
2020-07-24	Sarepta Therapeutics, Inc.	Sarepta Therapeutics Receives Fast Track Designation for SRP-9001 Micro-Dystrophin Gene Therapy for the Treatment of Duchenne Muscular Dystrophy sarepta.com ↗
2020-06-15	Sarepta Therapeutics, Inc.	Sarepta Therapeutics Announces Positive Safety and Efficacy Data from the SRP-9001 Micro-Dystrophin Gene Therapy Trial Published in JAMA Neurology sarepta.com ↗
2019-12-23	Sarepta Therapeutics, Inc.	Sarepta Therapeutics Announces Partnership with Roche in Territories Outside the United States for its Investigational Micro-dystrophin Gene Therapy for Duchenne Muscular Dystrophy, SRP-9001 sarepta.com ↗

Evidence & citations 8 cited values

Every value below carries the sentence it was read from. 12 sources stand behind the page.

Field	Value	Cited text
Known as	delandistrogene moxeparvovec	ClinicalTrials.gov intervention name — accepted as the source's own label NCT03375164 ↗ 8 NCT06128564 ↗ NCT05881408 ↗ NCT05967351 ↗ NCT06241950 ↗ NCT03769116 ↗ NCT06597656 ↗ NCT05096221 ↗ NCT04626674 ↗
Known as	Delandistrogene moxeparvovec component of elevidys	ChEMBL registry synonym — accepted as the source's own label CHEMBL4650241 ↗
Known as	ELEVIDYS	ClinicalTrials.gov intervention name — accepted as the source's own label NCT07542314 ↗
Known as	SRP-9001	ChEMBL registry synonym — accepted as the source's own label CHEMBL4650241 ↗
Action	Restore	“delivers a transgene encoding delandistrogene moxeparvovec micro-dystrophin, an engineered, functional form of dystrophin” PMID 39747998 ↗ Jan 2025
Modality	Gene therapy (AAV / viral vector)	“Delandistrogene moxeparvovec is an rAAVrh74 vector-based gene transfer therapy” PMID 39747998 ↗ Jan 2025
Route	Intravenous	“delandistrogene moxeparvovec intravenous (IV) administration” NCT03375164 ↗
Target	DMD	“Dystrophin exogenous gene” CHEMBL4650241 ↗