Drugs / delandistrogene moxeparvovec
Regulatory milestones approvals, filings & regulatory actions · 4 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed DMD-related muscular dystrophy 3 milestones | |||||
| Refused (EMA) | EU (EMA) | Elevidys | DMD-related muscular dystrophy | 2025-07-25 | “CHMP issued a negative opinion on the conditional marketing authorization (CMA) for ELEVIDYS (delandistrogene moxeparvovec) in ambulatory individuals ages three to seven years” sarepta.com ↗ “EMA’s CHMP issued an opinion not to recommend Elevidys™ (delandistrogene moxeparvovec) for the treatment of ambulatory individuals with Duchenne muscular dystrophy (DMD)” roche.com ↗ |
| Filed | US (FDA) | ELEVIDYS | DMD-related muscular dystrophy | 2024-02-16 | “today announced the U.S. Food and Drug Administration (FDA) has accepted and filed the Company's efficacy supplement to the Biologics License Application (BLA) for ELEVIDYS” sarepta.com ↗ “The U.S. Food and Drug Administration (FDA) has accepted and filed Sarepta’s efficacy supplement to the Biologics License Application (BLA) for ELEVIDYS” sarepta.com ↗ |
| Filed | US (FDA) | ELEVIDYS | DMD-related muscular dystrophy | 2023-12-22 | “today announced submission of an efficacy supplement to the Biologics License Application (BLA) for ELEVIDYS” sarepta.com ↗ “Submitted an efficacy supplement to the BLA for ELEVIDYS” sarepta.com ↗ |
| Rejected Indication not stated 1 milestone | |||||
| Refused (EMA) | EU (EMA) | Elevidys | — | 2025-09-24 | europa.eu ↗ |
Trials 9
| 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 Stop: Business |
| Phase 1 | NCT06241950 | Jan 2024 → Oct 2025 | Duchenne muscular dystrophy | Sarepta Therapeutics, Inc. | Terminated | No outcome recorded Stop: Business |
| 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 | Mixed |
| 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 · 1 missed | ||||||
| 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 | Missed primary |
News releases announcing trial results or a regulatory action · 44
| Date | Issuer | Release |
|---|---|---|
| 2026-09-30 | Sarepta Therapeutics, Inc. | Results Sarepta Therapeutics Presents New Data at 2026 World Muscle Society Annual Congress Demonstrating Safety and Clinically Meaningful Functional Benefit of ELEVIDYS (delandistrogene moxeparvovec) in Older Ambulatory Patients with Duchenne sarepta.com ↗
In ambulatory patients treated at ages 8 to 12, an age range in which functional decline becomes even more pronounced, treatment was associated with durable benefits across multiple measures over two years compared to a well-matched external control. |
| 2026-01-26 | Sarepta Therapeutics, Inc. | Results Sarepta Announces Positive Topline Three-Year EMBARK Results Showing ELEVIDYS Significantly Slows Disease Progression on Key Functional Measures in Ambulatory Duchenne Patients sarepta.com ↗
ELEVIDYS gene therapy demonstrated 70% or greater reduction in the rate of decline relative to the propensity-weighted external control group, as measured by Time to Rise (TTR) and 10-meter walk/run (10MWR). |
| 2025-11-25 | Sarepta Therapeutics, Inc. | Regulatory 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 ↗
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has approved dosing in Cohort 8 of ENDEAVOR (Study 9001-103). |
| 2025-11-14 | Sarepta Therapeutics, Inc. | Regulatory Sarepta Announces FDA’s Approval of Updated ELEVIDYS Prescribing Information sarepta.com ↗
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced an update to the prescribing information for ELEVIDYS (delandistrogene moxeparvovec-rokl), the only approved gene therapy for Duchenne muscular dystrophy (DMD). |
| 2025-09-04 | Chugai Pharmaceutical | Regulatory Implementation of Safety Measures for Elevidys chugai-pharm.co.jp ↗
We implemented these revisions to the electronic package insert on August 28, 2025. |
| 2025-08-01 | Hansa Biopharma AB | Results Hansa Biopharma announces supportive data from treatment with imlifidase prior to the administration of gene therapy for Duchenne muscular dystrophy hansabiopharma.com ↗
Twelve weeks after administration of the gene therapy, patients in the trial demonstrated evidence of AAV-mediated transduction and expression of micro-dystrophin, however with levels lower than seen in other trials with ELEVIDYS. |
| 2025-07-28 | Sarepta Therapeutics, Inc. | Regulatory 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 ↗
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that the U.S. Food and Drug Administration (FDA) notified Sarepta that it may lift its voluntary pause on shipments of ELEVIDYS (delandistrogene moxeparvovec) for ambulatory patients with Duchenne. |
| 2025-07-25 | Hoffmann-La Roche | Regulatory Roche provides regulatory update on Elevidys™ gene therapy for Duchenne muscular dystrophy in the EU roche.com ↗
EMA’s CHMP issued an opinion not to recommend Elevidys™ (delandistrogene moxeparvovec) for the treatment of ambulatory individuals with Duchenne muscular dystrophy (DMD) |
| 2025-07-25 | Sarepta Therapeutics, Inc. | Regulatory Sarepta Therapeutics Provides Clarifying Statement on ELEVIDYS sarepta.com ↗
Just before 6:00 p.m. ET today, the U.S. Food and Drug Administration (FDA) issued a press release announcing an investigation into the death of an eight-year-old Duchenne muscular dystrophy (Duchenne) patient who had received ELEVIDYS (delandistrogene moxeparvovec) gene therapy. |
| 2025-07-25 | Sarepta Therapeutics, Inc. | Regulatory Sarepta Therapeutics Acknowledges CHMP Negative Opinion for ELEVIDYS in the European Union sarepta.com ↗
the Committee for Medicinal Products for Human Use (CHMP) issued a negative opinion on the conditional marketing authorization (CMA) for ELEVIDYS |
| 2025-07-18 | Sarepta Therapeutics, Inc. | Regulatory Sarepta Therapeutics Provides Statement on ELEVIDYS sarepta.com ↗
Shortly after 2:30 p.m. ET today, Sarepta received an informal request from the U.S. Food and Drug Administration (FDA) to voluntarily halt shipment of ELEVIDYS (delandistrogene moxeparvovec), our gene therapy for Duchenne muscular dystrophy (Duchenne), in the U.S. |
| 2025-07-16 | Sarepta Therapeutics, Inc. | Regulatory Sarepta Therapeutics Announces Strategic Restructuring and Pipeline Prioritization Plan to Maintain Long-term, Sustainable Growth and Provides Update on ELEVIDYS Label sarepta.com ↗
U.S. FDA has requested and Sarepta has agreed to include a black box warning in the ELEVIDYS label, resolving any material issues with the ambulant population indication |
| 2025-06-16 | Chugai Pharmaceutical | Results Roche Announces New Safety Information of Elevidys for Non-Ambulatory Duchenne Muscular Dystrophy chugai-pharm.co.jp ↗
This new global safety information reports cases of acute liver failure in non-ambulatory DMD patients with fatal outcome. |
| 2025-06-15 | Hoffmann-La Roche | Regulatory [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 ↗
Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today new dosing restrictions, effective immediately, for ELEVIDYS™ (delandistrogene moxeparvovec), for non-ambulatory Duchenne muscular dystrophy (DMD) patients, irrespective of age, in both clinical and commercial settings. |
| 2025-05-21 | Sarepta Therapeutics, Inc. | Regulatory Sarepta Provides Update on UK Dosing in ENVISION Study of ELEVIDYS for the treatment of Duchenne Muscular Dystrophy sarepta.com ↗
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. |
| 2025-05-16 | Sarepta Therapeutics, Inc. | Results Sarepta Therapeutics Shares New Protein Expression and Safety Results from ENDEAVOR in Participants 2 Years Old at Time of Treatment sarepta.com ↗
Treatment with ELEVIDYS for Duchenne muscular dystrophy resulted in mean protein expression of 93.87% as measured by western blot in study participants (n=6) |
| 2025-05-16 | Sarepta Therapeutics, Inc. | Results Sarepta Therapeutics Presents Data at the American Society of Gene & Cell Therapy Conference, Including Statistically Significant Functional Outcomes for 8- and 9-Year-Old Patients in New Data Analysis of EMBARK Part 2 sarepta.com ↗ |
| 2025-05-13 | Chugai Pharmaceutical | Regulatory Chugai Receives Regulatory Approval for “ELEVIDYS” as a Gene Therapy Product for Duchenne Muscular Dystrophy in Japan chugai-pharm.co.jp ↗ |
| 2025-05-13 | Sarepta Therapeutics, Inc. | Regulatory Sarepta Therapeutics Announces Approval in Japan of ELEVIDYS, a Gene Therapy to Treat Duchenne Muscular Dystrophy sarepta.com ↗ |
| 2025-04-04 | Sarepta Therapeutics, Inc. | Regulatory Sarepta Therapeutics Provides Update on ELEVIDYS sarepta.com ↗
While the analysis is being finalized, recruitment and dosing in certain clinical studies of ELEVIDYS are temporarily halted. |
| 2025-03-17 | Hoffmann-La Roche | Results Roche to share latest scientific advancements from its neuromuscular portfolio at Muscular Dystrophy Association (MDA) 2025 conference roche.com ↗
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) |
| 2025-01-27 | Hoffmann-La Roche | Results Roche announces new results from EMBARK demonstrating significant sustained benefits of Elevidys in ambulatory individuals with Duchenne muscular dystrophy (DMD) roche.com ↗
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. |
| 2025-01-27 | Chugai Pharmaceutical | Results Roche Announces New Results from EMBARK Study of Elevidys in Duchenne Muscular Dystrophy chugai-pharm.co.jp ↗
Roche announces new results from EMBARK demonstrating significant sustained benefits of Elevidys in ambulatory individuals with Duchenne muscular dystrophy (DMD) |
| 2025-01-27 | Sarepta Therapeutics, Inc. | Results 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 ↗
Crossover-treated patients, those who received a placebo in Part 1 and crossed over at 52 weeks and were treated with ELEVIDYS in Part 2, improved 2.34 points from baseline compared to matched external controls on the North Star Ambulatory Assessment (NSAA) 52 weeks after treatment (P<0.0001), during which time the study remained blinded. |
| 2024-08-14 | Chugai Pharmaceutical | Regulatory Chugai Files Elevidys (SRP-9001) as a Gene Therapy Product for Duchenne Muscular Dystrophy in Japan chugai-pharm.co.jp ↗
The application will be reviewed under priority review in Japan |
| 2024-06-20 | Sarepta Therapeutics, Inc. | Regulatory Sarepta Therapeutics Announces Expanded US FDA Approval of ELEVIDYS to Duchenne Muscular Dystrophy Patients Ages 4 and Above sarepta.com ↗
Confirming the functional benefits, the FDA granted traditional approval for ambulatory patients. |
| 2024-02-28 | Sarepta Therapeutics, Inc. | Regulatory Sarepta Therapeutics Announces Fourth Quarter and Full-Year 2023 Financial Results and Recent Corporate Developments sarepta.com ↗
The U.S. Food and Drug Administration (FDA) has accepted and filed Sarepta’s efficacy supplement to the Biologics License Application (BLA) for ELEVIDYS (delandistrogene moxeparvovec-rokl). |
| 2024-02-16 | Sarepta Therapeutics, Inc. | Regulatory Sarepta Therapeutics Announces U.S. FDA Acceptance of an Efficacy Supplement to Expand the ELEVIDYS Indication sarepta.com ↗
today announced the U.S. Food and Drug Administration (FDA) has accepted and filed the Company's efficacy supplement to the Biologics License Application (BLA) for ELEVIDYS (delandistrogene moxeparvovec-rokl) (the “Efficacy Supplement”). |
| 2023-12-22 | Sarepta Therapeutics, Inc. | Regulatory 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 ↗
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced submission of an efficacy supplement to the Biologics License Application (BLA) for ELEVIDYS (delandistrogene moxeparvovec-rokl) to expand its labeled indication as follows “[ELEVIDYS is indicated for] the treatment of Duchenne muscular dystrophy (DMD) patients with a confirmed mutation in the DMD gene.” |
| 2023-11-01 | Sarepta Therapeutics, Inc. | Results Sarepta Therapeutics Announces Third Quarter 2023 Financial Results and Recent Corporate Developments sarepta.com ↗
ELEVIDYS-treated patients showed an increase on the North Star Ambulatory Assessment, a measure of motor function, compared to placebo-treated patients at 52 weeks, although the primary endpoint was not met. |
| 2023-10-31 | Chugai Pharmaceutical | Results Roche Announces Outcomes for EMBARK Study of Elevidys in Duchenne Muscular Dystrophy chugai-pharm.co.jp ↗
Roche announces EMBARK trial in Duchenne muscular dystrophy (DMD) did not reach primary endpoint, but shows positive efficacy outcomes on all timed functional key endpoints |
| 2023-10-30 | Hoffmann-La Roche | Results [Ad hoc announcement pursuant to Art. 53 LR] Roche announces EMBARK trial in Duchenne muscular dystrophy (DMD) did not reach primary endpoint, but shows positive efficacy outcomes on all timed functional key endpoints roche.com ↗
Elevidys-treated boys aged 4-7 years with Duchenne showed an increase on the North Star Ambulatory Assessment (NSAA), a measure of motor function, compared to placebo at 52 weeks but the primary endpoint was not met |
| 2023-10-30 | Sarepta Therapeutics, Inc. | Results Sarepta Therapeutics Announces Topline Results from EMBARK, a Global Pivotal Study of ELEVIDYS Gene Therapy for Duchenne Muscular Dystrophy sarepta.com ↗
In EMBARK, participants treated with ELEVIDYS (delandistrogene moxeparvovec-rokl) showed an increase on the North Star Ambulatory Assessment, a measure of motor function, compared to placebo-treated patients at 52 weeks, although the primary endpoint was not met |
| 2023-06-22 | Sarepta Therapeutics, Inc. | Regulatory Sarepta Therapeutics Announces FDA Approval of ELEVIDYS, the First Gene Therapy to Treat Duchenne Muscular Dystrophy sarepta.com ↗
This indication is approved under accelerated approval based on expression of ELEVIDYS micro-dystrophin observed in patients treated with ELEVIDYS. |
| 2023-05-24 | Sarepta Therapeutics, Inc. | Regulatory Sarepta Therapeutics Announces Update on Regulatory Review of SRP-9001 sarepta.com ↗
The FDA has also informed Sarepta that it requires modest additional time to complete the review, including final label negotiations and postmarketing commitment discussions, and that it anticipates that the review will be complete by June 22, 2023. |
| 2023-05-12 | Sarepta Therapeutics, Inc. | Regulatory Sarepta Therapeutics Announces Positive Vote from U.S. FDA Advisory Committee Meeting for SRP-9001 Gene Therapy to Treat Duchenne Muscular Dystrophy sarepta.com ↗
The committee’s positive vote is based on the evaluation of the totality of evidence including the SRP-9001 product design as well as biological and empirical data. |
| 2023-03-16 | Sarepta Therapeutics, Inc. | Regulatory Sarepta Therapeutics Announces Advisory Committee Meeting will be Held for SRP-9001 sarepta.com ↗
FDA’s decision to hold a public advisory committee meeting on the SRP 9001 BLA is a change from the communicated position at the midcycle meeting. |
| 2023-02-28 | Sarepta Therapeutics, Inc. | Regulatory Sarepta Therapeutics Announces Fourth Quarter and Full-Year 2022 Financial Results and Recent Corporate Developments sarepta.com ↗
Mid-cycle meeting complete; FDA does not plan to hold advisory committee for SRP-9001 |
| 2022-11-28 | Sarepta Therapeutics, Inc. | Regulatory 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 ↗
SRP-9001 has been granted Priority Review by the FDA, with a regulatory action date of May 29, 2023. |
| 2022-10-05 | Hoffmann-La Roche | Results Roche to present new data from its expanding neuromuscular disease portfolio at World Muscle Society 2022 roche.com ↗
The enduring response and safety observed in the study support the continuation of clinical trials assessing delandistrogene moxeparvovec using single dose gene therapy in patients with DMD. |
| 2022-09-29 | Sarepta Therapeutics, Inc. | Regulatory Sarepta Therapeutics Submits Biologics License Application for SRP-9001 for the Treatment of Ambulant Patients with Duchenne Muscular Dystrophy sarepta.com ↗
today announced that it has submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for the accelerated approval of SRP-9001 (delandistrogene moxeparvovec) to treat ambulant patients with Duchenne muscular dystrophy. |
| 2022-07-06 | Sarepta Therapeutics, Inc. | Results Sarepta Therapeutics’ Investigational Gene Therapy SRP-9001 for Duchenne Muscular Dystrophy Demonstrates Significant Functional Improvements Across Multiple Studies sarepta.com ↗
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today shared new functional data across multiple studies from the clinical development program for SRP-9001 (delandistrogene moxeparvovec) for the treatment of Duchenne muscular dystrophy. |
| 2022-03-08 | Hoffmann-La Roche | Results Roche to present new Evrysdi data at MDA 2022 and highlight expanding neuromuscular disease portfolio roche.com ↗
The results show an acceptable safety profile, and all patients demonstrated a clinically meaningful improvement in their North Star Ambulatory Assessment (NSAA) from baseline. |
| 2022-01-10 | Sarepta Therapeutics, Inc. | Results 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 ↗
SRP-9001-treated participants from the placebo crossover group (n=20, aged 5-8 at time of dosing SRP-9001) scored a statistically significant 2.0 points higher on the mean North Star Ambulatory Assessment* at 48 weeks compared to propensity-score weighted external controls** (p value=0.0009). |
All press releases naming this drug 60 releases
Evidence & citations 7 cited values
Every value below carries the sentence it was read from. 11 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | delandistrogene moxeparvovec | ClinicalTrials.gov intervention name — accepted as the source's own label NCT05881408 ↗ |
| Known as | Delandistrogene moxeparvovec component of elevidys | ChEMBL registry synonym — accepted as the source's own label CHEMBL4650241 ↗ |
| 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 ↗ |