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Gefurulimab

Phase 3

Generalized Myasthenia Gravis | Monoclonal antibody | Neurology |AstraZeneca PLC|Last Updated: Apr 1, 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
UNCONTROLLED
Total Trials1
Total Enrollment12
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06607627PK, PD, Safety, and Efficacy Study of Gefurulimab in Pediatric Patients With AChR+ Generalized Myasthenia GravisPHASE3 RECRUITING 12 — — Nov 13, 2024Jan 23, 2029Apr 1, 202613 United States, Brazil +3
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Study Endpoints
Primary Endpoints
Maximum Observed Serum Concentration (Cmax) of Gefurulimab
Day 1 predose through Week 18 predose
Serum Free Complement Component 5 (C5) Concentration
Day 1 predose through Week 18 predose
Secondary Endpoints
Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs)
Day 1 through Week 134
Change in Quantitative Myasthenia Gravis Score for Disease Severity (QMG) Total Score From Baseline Through Week 18
Baseline through Week 18
Number of Participants With ≥ 5-point Reduction From Baseline in the QMG Total Score Through Week 18
Baseline through Week 18
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
GefurulimabEXPERIMENTALOn Day 1, participants will receive a weight based loading dose followed by a weekly maintenance dose for up to 122 weeks.
Interventions
NameTypeDescription
GefurulimabCOMBINATION_PRODUCTCombination product consisting of syringe prefilled with gefurulimab will be administered weekly via subcutaneous (SC) injection.
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Eligibility Criteria
Age Range6 Years — 17 Years
SexALL
Healthy VolunteersNo
Study Sites13

Inclusion Criteria: United States of America (USA) specific inclusion criterion: * Participant must be 12 to \< 18 years of age at the time of signing the informed consent/assent. * All participants must be vaccinated against meningococcal infection from serogroups A, B, C, W, and Y within 3 years...

Countries:United StatesBrazilJapanPolandTaiwan
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT06607627primaryCompletionDate: changed
LOWMay 24, 2026NCT06607627studyFirstPostDate: changed