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fordadistrogene movaparvovec (fordadistrogene-movaparvovec)
Fordadistrogene movaparvovec delivers a functional dystrophin gene via AAV9 vector to restore muscle protein expression in Duchenne muscular dystrophy.
Fordadistrogene movaparvovec (PF-06939926) is a gene therapy developed by Pfizer utilizing an adeno-associated viral (AAV9) vector to deliver a functional dystrophin gene for treatment of Duchenne muscular dystrophy (DMD). The therapy represents a first-in-class approach to DMD by addressing the underlying genetic defect through in vivo gene delivery, targeting early-stage disease where muscle function remains partially preserved. Clinical development included Phase 1b, Phase 2, and Phase 3 trials enrolling approximately 154 patients across multiple cohorts; however, the program was discontinued after Phase 3 initiation, with one Phase 3 trial remaining active but not recruiting as of the latest data. A 2025 publication in Molecular Therapy reported initial cardiac safety observations from the Phase 1b trial, suggesting the therapy was generally well-tolerated in the studied population. The discontinuation reflects competitive pressures in the DMD gene therapy space and potential efficacy or manufacturing challenges. Pfizer's decision to halt development represents a significant setback in the AAV-based DMD therapeutic landscape, which includes competing programs from Sarepta Therapeutics and other biotechnology firms.
At a glance
| Generic name | fordadistrogene-movaparvovec |
|---|---|
| Sponsor | Pfizer Inc. |
| Drug class | Gene therapy; In vivo gene delivery |
| Target | Dystrophin gene (DMD); AAV9 receptor (AAVR) |
| Modality | Gene therapy |
| Therapeutic area | Rare Disease |
| Phase | discontinued |
Mechanism of action
Fordadistrogene movaparvovec is a gene therapy that uses an adeno-associated virus serotype 9 (AAV9) as a delivery vehicle to introduce a functional copy of the dystrophin gene into muscle cells. In Duchenne muscular dystrophy, mutations in the DMD gene prevent production of the dystrophin protein, which normally provides structural support to muscle fibers. Without dystrophin, muscle cells become damaged and progressively degenerate, leading to severe weakness and loss of function. By delivering a working copy of the gene directly into muscle tissue via the AAV9 vector, the therapy aims to restore dystrophin protein production and halt or slow muscle degeneration. The AAV9 vector is chosen for its ability to cross the blood-brain barrier and transduce muscle tissue efficiently, making it suitable for systemic delivery in a single administration.
Approved indications
Pipeline indications
- Duchenne Muscular Dystrophy (early-stage) — Phase 3
Common side effects
Key clinical trials
- Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy (PHASE3)
- Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy (PHASE2)
- A Study to Understand the Long-term Safety and Effects of an Experimental Gene Therapy for Duchenne Muscular Dystrophy. (PHASE3)
- A Study to Evaluate the Safety and Tolerability of PF-06939926 Gene Therapy in Duchenne Muscular Dystrophy (PHASE1)
- A Study to Evaluate AAV9 Neutralizing Antibody Seroconversion in Household Contacts.
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- fordadistrogene movaparvovec CI brief — competitive landscape report
- fordadistrogene movaparvovec updates RSS · CI watch RSS
- Pfizer Inc. portfolio CI