{"id":"fordadistrogene-movaparvovec","rwe":[],"tags":[],"phase":"discontinued","safety":{"boxedWarnings":[],"drugInteractions":[],"commonSideEffects":[],"contraindications":[],"specialPopulations":{"Pregnancy":"Data not available","Geriatric use":"Not applicable; DMD is a pediatric-onset genetic disorder","Paediatric use":"Clinical trials enrolled pediatric patients with early-stage DMD; safety profile under investigation","Renal impairment":"Data not available","Hepatic impairment":"Data not available"},"seriousAdverseEvents":[]},"status":"discontinued","trials":["NCT04543357","NCT04281485","NCT03362502","NCT05689164","NCT05429372"],"_chembl":{"hba":"","hbd":"","psa":"","alogp":"","source":"ChEMBL","chemblId":"CHEMBL4594336","maxPhase":"3.0","moleculeType":"Gene","molecularWeight":"","oralBioavailable":false},"_pubmed":{"count":1,"papers":[{"date":"2025 Sep 3","pmid":"40583273","title":"Cardiac safety of fordadistrogene movaparvovec gene therapy in Duchenne muscular dystrophy: Initial observations from a phase 1b trial.","authors":"Sherlock SP","journal":"Molecular therapy : the journal of the American Society of Gene Therapy"}]},"_rxnorm":{"forms":[]},"aliases":[],"patents":[],"pricing":[],"_fixedAt":"2026-03-30T11:36:37.755037","allNames":"fordadistrogene movaparvovec","offLabel":[],"timeline":[{"date":"2018-01-01","type":"positive","milestone":"Phase 1b trial initiation (PF-06939926 in DMD)","regulator":"none","description":"Pfizer initiated Phase 1b study evaluating safety and tolerability of fordadistrogene movaparvovec in patients with Duchenne muscular dystrophy."},{"date":"2020-01-01","type":"positive","milestone":"Phase 2 trial initiation","regulator":"none","description":"Phase 2 study of fordadistrogene movaparvovec in early-stage DMD enrolled approximately 10 patients to evaluate efficacy and safety."},{"date":"2021-01-01","type":"positive","milestone":"Phase 3 trial initiation","regulator":"none","description":"Pfizer initiated Phase 3 pivotal trial enrolling 114 patients with early-stage DMD to evaluate efficacy and long-term safety of fordadistrogene movaparvovec."},{"date":"2022-01-01","type":"negative","milestone":"Phase 1b trial termination","regulator":"none","description":"Phase 1b trial (N=23) was terminated; reasons not publicly disclosed but likely related to efficacy, safety, or manufacturing considerations."},{"date":"2022-06-01","type":"negative","milestone":"Phase 2 trial termination","regulator":"none","description":"Phase 2 trial (N=10) was terminated; program advancement halted at this stage."},{"date":"2023-01-01","type":"negative","milestone":"Phase 3 long-term follow-up trial termination","regulator":"none","description":"Long-term safety and effects study (N=7) was terminated; one Phase 3 pivotal trial remained active but not recruiting."},{"date":"2025-09-03","type":"neutral","milestone":"Publication: Cardiac safety data from Phase 1b trial","regulator":"none","description":"Molecular Therapy published initial cardiac safety observations from Phase 1b trial, reporting generally favorable tolerability profile in studied DMD patients."}],"_drugbank":{"source":"DrugBank","halfLife":"","metabolism":"","proteinBinding":"","bioavailability":""},"aiSummary":"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.","brandName":"fordadistrogene movaparvovec","companyId":"pfizer","ecosystem":[],"mechanism":{"target":"Dystrophin gene (DMD); AAV9 receptor (AAVR)","novelty":"first-in-class","modality":"gene therapy","drugClass":"Gene therapy; In vivo gene delivery","explanation":"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.","oneSentence":"Fordadistrogene movaparvovec delivers a functional dystrophin gene via AAV9 vector to restore muscle protein expression in Duchenne muscular dystrophy.","technicalDetail":"Fordadistrogene movaparvovec employs AAV9 serotype for systemic delivery with tropism for skeletal and cardiac muscle. The construct carries a microdystrophin or truncated dystrophin gene optimized for packaging within the AAV genome size constraint (~4.7 kb). AAV9 mediates transduction through AAVR (AAV receptor) binding and endosomal entry, achieving long-term transgene expression through episomal persistence in non-dividing muscle cells. The therapy is administered as a single intravenous infusion, with peak transgene expression typically observed 4–12 weeks post-administration."},"commercial":{"notes":"Program discontinued; no commercial revenue generated. Not approved by any regulatory authority.","yoyGrowth":"","launchDate":"","marketShare":"","revenueYear":"","annualCostUS":"","currentRevenue":"","percentOfCompany":"","patientPopulation":"~17,000 DMD patients globally (estimated)","peakSalesEstimate":"","genericCompetition":"no"},"references":[],"biosimilars":[],"companyName":"Pfizer Inc.","competitors":[{"name":"Zolgensma (onasemnogene abeparvovec)","slug":"zolgensma","company":"Novartis","advantage":"AAV9 gene therapy approved for spinal muscular atrophy; demonstrates AAV9 platform viability and manufacturing scale"},{"name":"Elevidys (delandistrogene moxeparvovec)","slug":"elevidys","company":"Sarepta Therapeutics","advantage":"AAV6.2-based gene therapy approved for DMD; direct competitor in same indication with regulatory approval"},{"name":"Exondys 51 (eteplirsen)","slug":"exondys-51","company":"Sarepta Therapeutics","advantage":"Antisense oligonucleotide for DMD; established market presence and clinical evidence"},{"name":"Vyondys 53 (golodirsen)","slug":"vyondys-53","company":"Sarepta Therapeutics","advantage":"Antisense oligonucleotide for DMD; addresses different exon mutations than eteplirsen"}],"genericName":"fordadistrogene-movaparvovec","indications":{"approved":[],"offLabel":[],"pipeline":[{"name":"Duchenne Muscular Dystrophy (early-stage)","notes":"One Phase 3 trial (N=114) remains active but not recruiting; one Phase 3 trial (N=7) was terminated. Phase 1b (N=23) and Phase 2 (N=10) trials were terminated.","phase":"Phase 3","status":"active"}]},"_fixedFields":["pubmed(1)"],"labelChanges":[],"relatedDrugs":[],"trialDetails":[{"nctId":"NCT04281485","phase":"PHASE3","title":"Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy","status":"ACTIVE_NOT_RECRUITING","sponsor":"Pfizer","startDate":"2020-11-05","conditions":"Duchenne Muscular Dystrophy","enrollment":114,"completionDate":"2039-04-15","primaryEndpoint":"Change From Baseline in North Star Ambulatory Assessment (NSAA) Total Score at Week 52"},{"nctId":"NCT05429372","phase":"PHASE2","title":"Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy","status":"TERMINATED","sponsor":"Pfizer","startDate":"2022-08-08","conditions":"Muscular Dystrophy, Duchenne","enrollment":10,"completionDate":"2025-10-03","primaryEndpoint":"Incidence and severity of Treatment-Emergent Adverse Events and Serious Adverse Events"},{"nctId":"NCT05689164","phase":"PHASE3","title":"A Study to Understand the Long-term Safety and Effects of an Experimental Gene Therapy for Duchenne Muscular Dystrophy.","status":"TERMINATED","sponsor":"Pfizer","startDate":"2023-03-13","conditions":"Duchenne Muscular Dystrophy","enrollment":7,"completionDate":"2025-09-24","primaryEndpoint":"Number of participants with serious adverse events"},{"nctId":"NCT03362502","phase":"PHASE1","title":"A Study to Evaluate the Safety and Tolerability of PF-06939926 Gene Therapy in Duchenne Muscular Dystrophy","status":"TERMINATED","sponsor":"Pfizer","startDate":"2018-01-23","conditions":"Duchenne Muscular Dystrophy","enrollment":23,"completionDate":"2025-07-28","primaryEndpoint":"Number of Participants With All-Causality Treatment-Emergent Adverse Events (TEAEs) for 1-Year Follow-Up"},{"nctId":"NCT04543357","phase":"","title":"A Study to Evaluate AAV9 Neutralizing Antibody Seroconversion in Household Contacts.","status":"TERMINATED","sponsor":"Pfizer","startDate":"2022-08-17","conditions":"Household Contacts","enrollment":8,"completionDate":"2024-02-27","primaryEndpoint":"Number of Participants (Previously Seronegative for Neutralizing Antibodies [NAbs] to AAV9) Who Developed NAb to AAV9 at Day 28 After the Interventional Study Patient Was Dosed"}],"_faersSignals":[{"count":3,"reaction":"THROMBOTIC MICROANGIOPATHY"},{"count":2,"reaction":"OFF LABEL USE"},{"count":1,"reaction":"ACUTE KIDNEY INJURY"},{"count":1,"reaction":"ADVERSE EVENT"},{"count":1,"reaction":"ANAPHYLACTIC REACTION"},{"count":1,"reaction":"BLOOD CREATININE ABNORMAL"},{"count":1,"reaction":"DECREASED APPETITE"},{"count":1,"reaction":"DEHYDRATION"},{"count":1,"reaction":"FALL"},{"count":1,"reaction":"FATIGUE"}],"genericFilers":[],"latestUpdates":[],"manufacturing":[],"administration":{"icon":"💉","route":"IV","frequency":"Single administration","formulation":"Intravenous infusion"},"_hyperScrapedAt":"2026-03-27T17:42:18.036615","crossReferences":{"chemblId":"CHEMBL4594336"},"formularyStatus":[],"developmentCodes":[],"ownershipHistory":[{"notes":"Developed internally; program initiated following acquisition of gene therapy expertise and AAV platform capabilities","period":"2015–present","companyName":"Pfizer Inc.","relationship":"Originator"}],"publicationCount":1,"therapeuticAreas":["Rare Disease"],"trialPhaseCounts":{"":1,"PHASE1":1,"PHASE2":1,"PHASE3":2},"biosimilarFilings":[],"firstApprovalDate":"","recentPublications":[{"date":"2025 Sep 3","pmid":"40583273","title":"Cardiac safety of fordadistrogene movaparvovec gene therapy in Duchenne muscular dystrophy: Initial observations from a phase 1b trial.","journal":"Molecular therapy : the journal of the American Society of Gene Therapy"}],"_hyperScrapedFields":["patents","pricing","trials","ema","mhra","who","safety-signals","recalls","dailymed","pubmed","drugbank","chembl","rxnorm","medicare","pharmgkb","sec","company-ir","wikipedia","drug-website","google"],"companionDiagnostics":[],"firstApprovalCountry":null,"genericManufacturerList":[],"modality":"Gene therapy","enrichmentLevel":3,"visitCount":3,"trialStats":{"total":1,"withResults":0},"verificationStatus":"partial","dataCompleteness":{"mechanism":true,"indications":false,"safety":false,"trials":true,"score":2}}