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Efgartigimod or Efgartigimod PH20

Phase 2

Generalized Myasthenia Gravis | Monoclonal antibody | Neurology |argenx SE|Last Updated: Apr 23, 2026

Target and mechanism

Molecular targetFCGRT
Target classAntagonist
ModalityMonoclonal antibody

Also known as Efgartigimod IV or Efgartigimod PH20 SC

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLEDDMC
Total Trials1
Total Enrollment12

FDA Designations

No designations recorded

Clinical trial landscape

Efgartigimod or Efgartigimod PH20 · 1 trial · 2 indications

Phase 2 1
NCT05374590Evaluating Long-term Safety of Efgartigimod Administered Intravenously and Efgartigimod PH20 Administered Subcutaneously in Children With Generalized Myasthenia GravisGeneralized Myasthenia Gravis
ENROLLING BY_INVITATION12 Analytics
PHASE2ENROLLING BY_INVITATION
Evaluating Long-term Safety of Efgartigimod Administered Intravenously and Efgartigimod PH20 Administered Subcutaneously in Children With Generalized Myasthenia Gravis
Generalized Myasthenia GravisUnlock trial analytics

Study Endpoints

Primary Endpoints

Incidence of AEs, SAEs and AESIs
Up to 4 years

AE : adverse event; SAE : serious adverse event; AESI : adverse event of special interest

Secondary Endpoints

Incidence of ADAs against efgartigimod
Up to 4 years
Incidence of antibodies against rHuPH20
Up to 4 years
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Efgartigimod or Efgartigimod PH20 SCEXPERIMENTALPatients receiving Efgartigimod IV treatment or Efgartigimod PH 20 SC treatment

Interventions

NameTypeDescription
Efgartigimod IV or Efgartigimod PH20 SCBIOLOGICALIntravenous infusion of Efgartigimod or Subcutaneous injection of Efgartigimod PH20 SC
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Eligibility Criteria

Age Range2 Years to 18 Years
SexALL
Healthy VolunteersNo
Study Sites16

Inclusion Criteria: * The participant completed ARGX-113-2006, defined as: 1. The participant reached End of Trial in trial ARGX-113-2006 or End of Study in ARGX-113-2207 and agreed to participate in the ARGX-113-2008 trial. 2. The participant qualifies for retreatment in trial ARGX-113-2006, ...

Countries:United StatesBelgiumCanadaFranceGermanyIsraelNetherlandsPolandSpainUnited Kingdom
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Frequently asked questions about Efgartigimod or Efgartigimod PH20

What is Efgartigimod used for?

Efgartigimod, also known as Efgartigimod PH20, is an investigational monoclonal antibody being studied for the treatment of generalized Myasthenia Gravis (gMG) in children. It is currently in Phase 2 clinical development and is not yet approved by regulatory authorities.

What does Efgartigimod target?

Efgartigimod targets the neonatal Fc receptor (FcRn). By blocking this receptor, it reduces levels of pathogenic IgG antibodies that contribute to the autoimmune attack in generalized Myasthenia Gravis. This mechanism is being evaluated in an ongoing Phase 2 clinical trial.

Who makes Efgartigimod?

Efgartigimod is being developed by argenx SE, a biopharmaceutical company traded on the NASDAQ under the ticker symbol ARGX. The company is conducting a Phase 2 clinical trial to evaluate the drug's safety and efficacy in pediatric patients with generalized Myasthenia Gravis.

What phase is Efgartigimod in?

Efgartigimod is currently in Phase 2 clinical development. It is an investigational drug and has not received FDA approval. The ongoing Phase 2 trial is designed to evaluate the long-term safety of Efgartigimod administered intravenously and Efgartigimod PH20 administered subcutaneously in children with generalized Myasthenia Gravis.

What clinical trials is Efgartigimod in?

Efgartigimod is being studied in a Phase 2 clinical trial registered as NCT05374590. This trial is evaluating the long-term safety of Efgartigimod administered intravenously and Efgartigimod PH20 administered subcutaneously in children with generalized Myasthenia Gravis. The trial is enrolling by invitation and aims to enroll 12 participants across multiple countries.

Is Efgartigimod the same as Efgartigimod PH20?

Yes, Efgartigimod and Efgartigimod PH20 are related forms of the same drug. Efgartigimod is administered intravenously, while Efgartigimod PH20 is a subcutaneous formulation. Both are being evaluated in the same Phase 2 clinical trial for the treatment of generalized Myasthenia Gravis in children.