argenx

argenx works on 18 rare diseases tracked on Trial Friend, including Amyotrophic Lateral Sclerosis, Bullous Pemphigoid, Chronic Inflammatory Demyelinating Polyneuropathy and 15 more, with 27 recruiting clinical trials and 2 FDA-approved rare disease drugs.

argenx is a global immunology company headquartered in Belgium, focused on FcRn-blocking antibody therapies for rare autoimmune diseases. Vyvgart (efgartigimod) is FDA-approved for generalized myasthenia gravis (2021) and chronic inflammatory demyelinating polyneuropathy (CIDP, April 2025). Vyvgart Hytrulo, a subcutaneous formulation, is also approved for both indications. The pipeline includes late-stage programs in immune thrombocytopenia, pemphigus, and myositis.

Type
Rare Disease Specialist
Ticker
ARGX
Headquarters
Zwijnaarde, Belgium
Founded
2008
Website
argenx.com
27
Active Rare Disease Trials
2
Approved Rare Disease Drugs
18
Rare Diseases in Portfolio
18
Years Active

Focus areas at argenx

As a rare disease specialist, argenx has active clinical trial programs and drug development efforts across 18 rare diseases, including Amyotrophic Lateral Sclerosis, Bullous Pemphigoid, Chronic Inflammatory Demyelinating Polyneuropathy, Dermatomyositis, Epidermolysis Bullosa, and 13 additional rare conditions. These programs may span orphan drug designation, novel therapeutic mechanisms, and precision medicine approaches targeting the underlying causes of each disease.

The clinical trials section below shows all active and recruiting studies sponsored by argenx, sourced live from ClinicalTrials.gov. Each trial includes its current recruitment status, study phase (Phase 1 through Phase 4), conditions under investigation, and the number of active trial sites. The FDA-approved drugs section lists treatments that have received U.S. Food and Drug Administration approval, with brand names, generic names, approval dates, and matched rare disease indications from the openFDA database.

argenx is headquartered in Zwijnaarde, Belgium, founded in 2008, publicly traded under the ticker symbol ARGX. The company dedicates its research and development resources exclusively to rare and orphan diseases, working to bring innovative treatments to patients with conditions that have historically had limited or no treatment options.

argenx Drug Pipeline

argenx has 27 active clinical trials across 4 development stages, with 27 currently recruiting participants. Clinical trials advance through phases: Phase 1 tests safety in a small group, Phase 2 evaluates effectiveness and side effects, Phase 3 confirms benefit in a larger population, and Phase 4 monitors long-term safety after FDA approval.

Note: This pipeline includes all of argenx's active interventional trials, not only those targeting rare diseases. We show the full pipeline because a company's broader research activity, therapeutic expertise, and development infrastructure directly shape its ability to advance rare disease programs. A strong overall pipeline often signals deeper clinical operations, faster enrollment capabilities, and greater commitment to bringing new treatments to patients.

Understand argenx's pipeline
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6
Phase 26 trials
13
Phase 313 trials
2
Phase 4 / Post-Market2 trials
6
Other6 trials
Recruiting
Chronic Inflammatory Demyelinating Polyradiculoneuropathy+2 more
Recruiting
Recruiting

argenx Clinical Trials (27)

Active and recruiting clinical trials sponsored by argenx, sourced live from ClinicalTrials.gov. Each trial card shows the study phase, current recruitment status, conditions under investigation, study locations, eligibility criteria, and a direct link to the full ClinicalTrials.gov record. You can also download a one-page PDF summary to share with your doctor.

Note: Recruitment statuses on ClinicalTrials.gov may not immediately reflect recent FDA decisions, sponsor announcements, or enrollment changes. Always confirm a trial's current status directly with the study coordinator.

Ask about argenx's trials
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RECRUITINGPHASE2, PHASE3Recently updatedNCT06392386

A Study of Efgartigimod PH20 SC in Children Between 2 and Less Than 18 Years of Age With Generalized Myasthenia Gravis

Intervention: Efgartigimod PH20 SC

The purpose of this study is to measure the pharmacokinetics (PK), pharmacodynamics (PD), safety, tolerability, and immunogenicity of efgartigimod PH20 SC in pediatric participants with gMG aged 2 to \<18 years. The primary goal is to confirm an appropriate dose of efgartigimod PH20 SC for pediatric patients using PK and PD results from this study. Participants will receive injections of efgartigimod PH20 SC and will be monitored for safety until the end of the study. At the end of the follow-up period, eligible participants may roll over to an open-label extension (OLE) study. The participants will be in the study for up to 14 weeks.

Ages 2 Years - 17 Years22 locations
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RECRUITINGPHASE2Recently updatedNCT06655155

A Study to Assess the Efficacy and Safety of Efgartigimod PH20 SC in Adults With Systemic Sclerosis

Intervention: Efgartigimod PH20 SC, Placebo PH20 SC

The main purpose of this study is to evaluate the effect and safety of efgartigimod PH20 SC compared to placebo in adults with systemic sclerosis. The study consists of a screening period, a treatment period of up to 48 weeks and a safety follow-up period. After the screening period, eligible participants will be randomized in a 2:1 ratio to receive either efgartigimod PH20 SC or placebo. The total study duration can be up to approximately 15 months. More information can be found on: https://clinicaltrials.argenx.com/esscape

Ages 18 Years+79 locations
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RECRUITINGPHASE2Recently updatedNCT07673627

ADAPT Forward 2 - ISA2 - a Study to Evaluate the Safety, Tolerability and Efficacy of Empasiprubart IV Monotherapy in Participants With AChR-Ab Seropositive Generalized Myasthenia Gravis

Intervention: Empasiprubart IV, Placebo IV, Efgartigimod PH20 SC PFS

This study is part of the ADAPT Forward platform study (NCT07294170). ADAPT Forward is a platform study with the aim to look at how safe different drugs are and how well they work for people with myasthenia gravis. The goal is to find the best therapeutic approach to reduce patients' side effects and improve their quality of life. The aim of this ISA2 is to investigate the effects of empasiprubart in participants with AChR-Ab seropositive generalized myasthenia gravis (gMG). The ADAPT Forward master protocol is registered on https://clinicaltrials.gov/study/NCT07294170 More information can be found here: https://clinicaltrials.argenx.com/adaptforward2

Ages 18 Years+4 locations
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RECRUITINGRecently updatedNCT07294170

ADAPT Forward - Master Protocol of a Platform Study to Evaluate the Safety and Efficacy of Multiple Regimens in Participants With Myasthenia Gravis

Intervention: Efgartigimod IV, Empasiprubart IV, Placebo IV, Efgartigimod PH20 SC PFS

ADAPT Forward is a platform study with the aim to look at how safe different drugs are and how well they work for people with myasthenia gravis. The goal is to find the best therapeutic approach to reduce patients' side effects and improve their quality of life.

Ages 18 Years+36 locations
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RECRUITINGPHASE3Recently updatedNCT06920004

A Study to Assess Efficacy and Safety of Empasiprubart Versus IVIg in Adults With CIDP

Intervention: empasiprubart, IVIg, empasiprubart-placebo, IVIg-placebo

The main purpose of this study is to compare empasiprubart and IVIg for treating people with CIDP. This study consists of a Part A where participants will either receive empasiprubart and a placebo resembling IVIg, or IVIg and a placebo resembling empasiprubart for 24 weeks (6 months). Following Part A, participants will enter Part B in which all participants will receive empasiprubart for 96 weeks (24 months). More information can be found here: https://clinicaltrials.argenx.com/emvigorate

Ages 18 Years+101 locations
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RECRUITINGPHASE2, PHASE3Recently updatedNCT07194850

A Study of Efgartigimod IV in Participants From 12 Years to Less Than 18 Years of Age With Chronic Immune Thrombocytopenia (ITP)

Intervention: Efgartigimod IV, Placebo IV

The main purpose of this study is to confirm the correct dose of efgartigimod IV for treating patients aged 12 to younger than 18 years with chronic immune thrombocytopenia (ITP). The study consists of a double-blinded treatment period (DBTP) in which the participants will be randomized in a 2:1 ratio to receive either efgartigimod IV or placebo IV. At the end of the treatment period (up to 24 weeks), all participants will receive efgartigimod IV during the first year open-label treatment period (OLTP1). At the end of the first OLTP1, participants may begin a second year (OLTP2). After the OLTP2, the participants will enter a follow-up period (approximately 8 weeks) while off study drug. The participants will be in the study for up to 138 weeks. More information can be found here: https://clinicaltrials.argenx.com/advancejunior

Ages 12 Years - 17 Years22 locations
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RECRUITINGPHASE2Recently updatedNCT07284420

ADAPT Forward 1 - ISA1 - a Study to Evaluate Empasiprubart IV as add-on Therapy to Efgartigimod IV in Participants With AChR-Ab Seropositive Generalized Myasthenia Gravis With a Partial Clinical Response to Efgartigimod

Intervention: Efgartigimod IV, Empasiprubart IV

This study is part of the ADAPT Forward platform study (NCT07294170). ADAPT Forward is a platform study with the aim to look at how safe different drugs are and how well they work for people with myasthenia gravis. The goal is to find the best therapeutic approach to reduce patients' side effects and improve their quality of life. The aim of this ISA1 is to evaluate the safety and therapeutic relevance of empasiprubart as add-on therapy to efgartigimod in participants with AChR-Ab seropositive generalized myasthenia gravis. The ADAPT Forward master protocol is registered on https://clinicaltrials.gov/study/NCT07294170 More information can be found here: https://clinicaltrials.argenx.com/adaptforward1

Ages 18 Years+33 locations
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argenx FDA-Approved Drugs (2)

Medications developed or marketed by argenx that have received U.S. Food and Drug Administration approval. Drug data is sourced from the openFDA database and includes brand names, generic names, approval dates, and matched rare disease indications. Where a drug treats a condition covered by Trial Friend, the disease name links directly to that disease page.

Drug NameBrand NameRare DiseasesApproval Date
EFGARTIGIMOD ALFA
Neonatal Fc Receptor Blocker [EPC]
VYVGART
intravenous
Dec 17, 2021
EFGARTIGIMOD ALFA AND HYALURONIDASE (HUMAN RECOMBINANT)
Endoglycosidase [EPC]
VYVGART Hytrulo
subcutaneous
Jun 20, 2023

argenx Trial Locations

argenx clinical trials are running at 1,509 sites in 59 countries. Click any country to drill down by state, city, and individual research facility. Proximity to a trial site is one of the most important factors in deciding whether to participate.

United States
289▼
Germany
128▼
Italy
108▼
China
106▼
Poland
100▼
Japan
91▼
Spain
61▼
Georgia
48▼
United Kingdom
47▼
France
43▼
Argentina
28▼
Romania
28▼

Rare Disease Focus Areas (18)

Diseases targeted by argenx's clinical trial and drug development programs

Amyotrophic Lateral SclerosisNeurological & Neuromuscular

Amyotrophic lateral sclerosis is a progressive neurodegenerative disease destroying motor neurons in the brain and spinal cord. This causes progressive weakness and paralysis of voluntary muscles whil...

Prevalence: About 5,000 new cases per year in the U.S.; approximately 16,000 Americans living with ALS at any given time
Bullous PemphigoidDermatologic

Bullous pemphigoid is an autoimmune blistering disorder caused by autoantibodies against basement membrane proteins, particularly BP180 and BP230. The condition leads to formation of large fluid-fille...

Prevalence: Approximately 2-23 per million people per year; incidence increases sharply with age
Chronic Inflammatory Demyelinating PolyneuropathyNeurological & Neuromuscular

Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) is an autoimmune disorder affecting the peripheral nerves, causing progressive weakness and impaired function in the legs and arms. The immune ...

Prevalence: Approximately 1 per 100,000 to 1 per 50,000 people; estimated 250,000 people have CIDP in North America
DermatomyositisAutoimmune & Inflammatory

Dermatomyositis is a rare autoimmune inflammatory disease causing muscle weakness and distinctive skin rashes, particularly over joints. It involves inflammation of muscles and skin blood vessels. Abo...

Prevalence: Approximately 1-10 cases per million people; juvenile-onset incidence about 0.4 cases per 100,000 children
Epidermolysis BullosaDermatologic

Epidermolysis bullosa is a group of rare genetic blistering disorders caused by mutations affecting proteins anchoring the epidermis to the dermis. Fragile skin blisters and erodes with minimal trauma...

Prevalence: 1 in 50,000 births; approximately 25,000-30,000 Americans affected
Graves’ DiseaseEndocrine & Hormonal

Graves' disease is the most common cause of hyperthyroidism, an autoimmune condition where antibodies stimulate the thyroid gland to produce excessive thyroid hormone. It can cause a wide range of sym...

Prevalence: Affects about 1 in 200 people; most common in women ages 30-50

Patient Resources

Organizations and resources related to argenx's rare disease focus areas

Frequently Asked Questions About argenx

Common questions about argenx's rare disease programs, clinical trials, and treatments.