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Gefurulimab

Phase 3

Generalized Myasthenia Gravis | Monoclonal antibody | Neurology |AstraZeneca PLC|Last Updated: Apr 1, 2026

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLED
Total Trials1
Total Enrollment12

FDA Designations

No designations recorded

Clinical trial landscape

Gefurulimab · 2 trials · 3 indications

Phase 3 1Phase 1 1
NCT06607627PK, PD, Safety, and Efficacy Study of Gefurulimab in Pediatric Patients With AChR+ Generalized Myasthenia GravisGeneralized Myasthenia Gravis
RECRUITING12 Analytics
PHASE3RECRUITING
PK, PD, Safety, and Efficacy Study of Gefurulimab in Pediatric Patients With AChR+ Generalized Myasthenia Gravis
Generalized Myasthenia Gravis
NCT0660762712
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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
Area Under the Plasma Concentration Versus Time Curve From Time 0 Extrapolated to Infinity (AUC0-inf) of Gefurulimab
Day 1 predose through Day 92 postdose

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 to 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 StatesBrazilJapanPolandTaiwanChina
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Frequently asked questions about Gefurulimab

What is Gefurulimab used for?

Gefurulimab is an investigational monoclonal antibody being developed for generalized myasthenia gravis (gMG), a neuromuscular disorder. It is also being studied in healthy participants to assess its pharmacokinetics. The drug is currently in Phase 3 clinical development for gMG and is not yet approved by regulatory authorities.

Who is developing Gefurulimab?

Gefurulimab is being developed by AstraZeneca PLC, a biopharmaceutical company listed on the stock exchange under the ticker AZN. The company is conducting clinical trials to evaluate the drug's safety and efficacy in patients with generalized myasthenia gravis.

What phase is Gefurulimab in?

Gefurulimab is in Phase 3 clinical development for generalized myasthenia gravis. It is also being studied in a Phase 1 trial in healthy participants. The drug is investigational and has not been approved by the FDA or other regulatory agencies.

What clinical trials is Gefurulimab in?

Gefurulimab is being studied in two clinical trials. NCT06607627 is a Phase 3 study in pediatric patients with AChR+ generalized myasthenia gravis, currently recruiting in the US, Brazil, Japan, Poland, and Taiwan. NCT06677138 is a completed Phase 1 pharmacokinetic study in healthy Chinese adult participants.

How does Gefurulimab work?

Gefurulimab is a monoclonal antibody, a type of -mab antibody. Its specific molecular target has not been disclosed in available information. The drug is being studied for its potential to treat generalized myasthenia gravis by modulating immune system components involved in the disease process.