Drugs / fordadistrogene movaparvovec
fordadistrogene movaparvovec
Trials 4
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 11 trial | ||||||
| Phase 1 | NCT03362502 | Jan 2018 → Mar 2022 | Duchenne muscular dystrophy | Pfizer | Terminated | No outcome recorded |
| Phase 21 trial | ||||||
| Phase 2 | NCT05429372 | Aug 2022 → Oct 2025 | Duchenne muscular dystrophy | Pfizer | Terminated | No outcome recorded |
| Phase 32 trials · 1 missed | ||||||
| Phase 3 | NCT05689164 | Mar 2023 → Sep 2025 | Duchenne muscular dystrophy | Pfizer | Terminated | No outcome recorded |
| Phase 3 | NCT04281485 | Nov 2020 → May 2024 overdue | Duchenne muscular dystrophy | Pfizer | Active not recruiting | Missed primary |
News releases announcing trial results or a regulatory action · 5
| Date | Issuer | Release |
|---|---|---|
| 2024-06-12 | Pfizer | Results Pfizer Provides Update on Phase 3 Study of Investigational Gene Therapy for Ambulatory Boys with Duchenne Muscular Dystrophy pfizer.com ↗
Key secondary endpoints, including 10-meter run/walk velocity and time to rise from floor velocity, also did not show a significant difference between participants treated with fordadistrogene movaparvovec and placebo. |
| 2020-10-01 | Pfizer | Regulatory Pfizer Receives FDA Fast Track Designation for Duchenne Muscular Dystrophy Investigational Gene Therapy pfizer.com ↗
Pfizer Inc. (NYSE: PFE) today announced that its investigational gene therapy candidate (PF-06939926) being developed to treat Duchenne muscular dystrophy (DMD) received Fast Track designation from the U.S. Food and Drug Administration (FDA). |
| 2020-05-15 | Pfizer | Results Pfizer’s New Phase 1b Results of Gene Therapy in Ambulatory Boys with Duchenne Muscular Dystrophy (DMD) Support Advancement into Pivotal Phase 3 Study pfizer.com ↗
Pfizer Inc. (NYSE: PFE) today announced updated Phase 1b clinical data on PF-06939926, an investigational gene therapy being developed to treat Duchenne muscular dystrophy (DMD). |
| 2019-07-01 | AskBio Inc | Results Statement: Pfizer Phase 1b Clinical Trial Results askbio.com ↗
On June 28, 2019, Pfizer reported results from a Phase 1b clinical trial of PF-06939926, its investigational gene therapy for Duchenne muscular dystrophy (DMD). |
| 2019-06-28 | Pfizer | Results Pfizer Presents Initial Clinical Data on Phase 1b Gene Therapy Study for Duchenne Muscular Dystrophy (DMD) pfizer.com ↗
Preliminary safety results show that the most common adverse events suspected to be related to PF-06939926 are nausea, vomiting, decreased appetite, tiredness and/or fever, which were reported within a few d |
All press releases naming this drug 5 releases
Evidence & citations 6 cited values
Every value below carries the sentence it was read from. 6 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | fordadistrogene movaparvovec | “Fordadistrogene movaparvovec is an investigational recombinant adeno-associated virus 9 (rAAV9)-based vector encoding a mini-dystrophin transgene protein for Duchenne muscular...” PMID 41722591 ↗ Mar 20262“Fordadistrogene movaparvovec (PF-06939926) is an adeno-associated virus serotype 9 gene therapy containing a miniaturized dystrophin being developed for DMD” PMID 40579547 ↗ Jun 2025 |
| Known as | Aav9.hck.opti-dys.delta.3978 | ChEMBL registry synonym — accepted as the source's own label CHEMBL4594336 ↗ |
| Known as | PF-06939926 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT03362502 ↗ |
| Action | Restore | “which aims to restore functional protein to muscle” PMID 40579547 ↗ Jun 2025 |
| Modality | Gene therapy (AAV / viral vector) | “Fordadistrogene movaparvovec (PF-06939926) is an adeno-associated virus serotype 9 gene therapy” PMID 40579547 ↗ Jun 2025 |
| Route | Intravenous | “a single intravenous low-dose (n = 3) or high-dose (n = 16) fordadistrogene movaparvovec” PMID 40579547 ↗ Jun 2025 |