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Efgartigimod Alfa

Phase 2

Optic Neuritis | Small molecule | Neurology |argenx SE|Last Updated: Nov 10, 2025

Target and mechanism

Molecular targetFCGRT
Target classAntagonist
ModalitySmall molecule

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLEDBiomarker
Total Trials1
Total Enrollment20

FDA Designations

No designations recorded

Clinical trial landscape

Efgartigimod Alfa · 1 trial · 1 indication

Phase 2 1
NCT06453694Efgartigimod for the Treatment of Acute Optic NeuritisOptic Neuritis
RECRUITING20 Analytics
PHASE2RECRUITING
Efgartigimod for the Treatment of Acute Optic Neuritis
Optic NeuritisUnlock trial analytics

Study Endpoints

Primary Endpoints

Recruitment Rate
2 years

Number of enrolled participants per month

Study Adherence Rate
2 years

Proportion of randomized participants who receive both doses of assigned study intervention, attend all assigned study visits, and complete at least the high contrast visual acuity, low contrast visual acuity, and Pelli-Robson assessments at all visits

Change in high contrast visual acuity for effect size and standard deviation estimation
1 month

Difference in change in high-contrast visual acuity from baseline to 1 month between groups

Change in low contrast visual acuity for effect size and standard deviation estimation
1 month

Difference in change in low contrast visual acuity (LCVA) (# of letters seen at 2.5% illumination) from baseline to 1 month between groups

Secondary Endpoints

Retention rate
2 years
Screen failure rate
2 years
Pre-screen failure rate
2 years
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Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Efgartigmod alfaACTIVE_COMPARATOR10 patients will receive efgartigimod alfa All participants will receive standard of care high dose corticosteroids for 3 days with a prednisone taper over 3 weeks.
PlaceboPLACEBO_COMPARATOR10 patients will receive placebo. All participants will receive standard of care high dose corticosteroids for 3 days with a prednisone taper over 3 weeks.

Interventions

NameTypeDescription
Efgartigimod AlfaDRUG2,016 mg will be administered subcutaneously by a healthcare provider on Day 0 and Day 3 of the trial. Rescue therapy with therapeutic plasma exchange will be given to any participant based on the results of Day 7 evaluation.
PlaceboDRUGSubcutaneous injection of placebo will be administered by a healthcare provider on Day 0 and Day 3 of the trial. Rescue therapy with therapeutic plasma exchange will be given to any participant based on the results of Day 7 evaluation.
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites2

Inclusion Criteria: 1. Provision of signed and dated informed consent form 2. Stated willingness to comply with all study procedures and availability for the duration of the study 3. Adults aged 18 years or older 4. Diagnosed with a first episode of optic neuritis, based on clinical presentation (i...

Countries:United States
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Frequently asked questions about Efgartigimod Alfa

What is Efgartigimod Alfa used for in Optic Neuritis?

Efgartigimod Alfa is being studied for the treatment of acute optic neuritis, a neurological condition involving inflammation of the optic nerve. It is currently in Phase 2 clinical development as an investigational therapy for this indication.

What does Efgartigimod Alfa target?

Efgartigimod Alfa targets the neonatal Fc receptor (FcRn), which plays a role in recycling IgG antibodies. By blocking FcRn, it reduces levels of pathogenic IgG antibodies, potentially mitigating the autoimmune attack on the optic nerve in optic neuritis.

Who is developing Efgartigimod Alfa?

Efgartigimod Alfa is being developed by argenx SE, a biopharmaceutical company listed on the NASDAQ under the ticker ARGX. The company is conducting a Phase 2 clinical trial for this drug in optic neuritis.

What phase is Efgartigimod Alfa in for optic neuritis?

Efgartigimod Alfa is in Phase 2 clinical development for optic neuritis. It is an investigational drug and has not been approved by regulatory authorities for this indication. The ongoing trial is recruiting participants in the United States.

What clinical trials is Efgartigimod Alfa in for optic neuritis?

Efgartigimod Alfa is being evaluated in a Phase 2, randomized, double-blind, placebo-controlled trial (NCT06453694) for acute optic neuritis. The study, titled 'Efgartigimod for the Treatment of Acute Optic Neuritis,' is recruiting 20 participants aged 18 years and older in the United States.

Is Efgartigimod Alfa the same as Vyvgart?

Efgartigimod Alfa is the active ingredient in Vyvgart, a brand name for the drug. In the context of optic neuritis, it is being investigated under the name Efgartigimod Alfa in a Phase 2 clinical trial.