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SRP-9003

Phase 3

Limb-girdle Muscular Dystrophy | Monoclonal antibody | Neurology |Sarepta Therapeutics, Inc.|Last Updated: Mar 2, 2026

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
UNCONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment17
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06246513A Trial to Learn More About an Experimental Gene Therapy Called Bidridistrogene Xeboparvovec (SRP-9003) as a Possible Treatment for Limb Girdle Muscular Dystrophy 2E/R4PHASE3 ACTIVE NOT_RECRUITING 17 — — Jan 15, 2024Nov 30, 2029Mar 2, 202610 United States, Belgium +4
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Study Endpoints
Primary Endpoints
Cohort 1: Change from Baseline in β-SG Expression at Day 60 Post-dose as Measured by Immunofluorescence (IF) Percent β-SG Positive Fibers
Baseline, Day 60
Secondary Endpoints
Cohort 1 and Cohort 2: Change From Baseline in β-SG Expression at Day 60 Post-dose as Measured by Western Assay
Baseline, Day 60
Cohort 1 and Cohort 2: Change From Baseline in β-SG Expression at Day 60 Post-dose as Measured by IF Percent Fluorescent Intensity (PFI)
Baseline, Day 60
Cohort 2: Change From Baseline in β-SG Expression at Day 60 Post-dose as Measured by IF Percent β-SG Positive Fibers (PβSGPF)
Baseline, Day 60
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
SRP-9003EXPERIMENTALParticipants will receive a single intravenous (IV) infusion of SRP-9003.
Interventions
NameTypeDescription
SRP-9003BIOLOGICALSolution for single IV infusion
GlucocorticoidDRUGOral tablet (prophylactic)
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Eligibility Criteria
Age Range4 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites10

Inclusion Criteria: * Cohort 1, only ambulatory participants: * Able to walk without assistive aid * 10MWR \<30 seconds * NSAD ≥25 * Cohort 2, only non-ambulatory participants: * 10MWR ≥30 seconds or unable to perform * PUL 2.0 entry scale score ≥3 * Participants must possess 1 homozygo...

Countries:United StatesBelgiumGermanyItalySpainUnited Kingdom
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT06246513primaryCompletionDate: changed
LOWMay 24, 2026NCT06246513studyFirstPostDate: changed