2024 In Brief
In 2024, we established our ‘Vision 2030’ outlining our long-term commitment to transform the treatment of severe autoimmune disease with VYVGART, empasiprubart and our expanding pipeline of antibody-based therapeutics. With our eyes set on 2030, we are targeting the treatment of 50,000 patients globally, securing 10 labeled indications across all approved medicines, and advancing five pipeline candidates into Phase 3 development.
In 2024, we made important progress to reach this goal. We grew our global commercial footprint in gMG to reach more than 10,000 patients and we remain on track to expand into additional regions throughout 2025. We received FDA approval for VYVGART HYTRULO for the treatment of CIDP and have been working hard to bring VYVGART HYTRULO to CIDP patients, reaching approximately 1,000 patients by the end of 2024. This, together with our continued growth in MG, translated in $2.2 billion in product net sales in 2024. We look forward to continued commercial execution as we expand our patient reach through label enabling studies in seronegative gMG and ocular MG patient populations and we continue to innovate on the patient experience with our pre-filled syringe (PFS) with VYVGART SC, with an PDUFA Date of April 10, 2025.
We made significant progress in evaluating efgartigimod across additional autoimmune diseases. We announced the GO-decisions for primary Sjögren’s disease (SjD) and three subsets of myositis (immune-mediated necrotizing myopathy (IMNM), anti-synthetase syndrome (ASyS), dermatomyositis (DM)), for which we are currently running Phase 3 clinical trials. We continue to evaluate efgartigimod in more than 10 additional indications, and this year, we added autoimmune encephalitis to the line-up. We are excited to add another indication in 2025.
In 2024 we made significant progress with our second asset, empasiprubart (ARGX-117) targeting complement component 2 (C2). empasiprubart has now shown proof-of-concept in multifocal motor neuropathy (MMN) and has started its first Phase 3 in this indication. We additionally announced CIDP as the 4th indication for which we go straight to a Phase 3 clinical trial, expected to start in 2025.
Beyond our first two assets, efgartigimod and empasiprubart, we worked to further advance our third clinical pipeline asset, ARGX-119, targeting muscle-specific kinase (MuSK). ARGX-119 has started its first proof-of-concept clinical trial in congenital myasthenic syndrome (CMS) and amyotrophic lateral sclerosis (ALS) this year and we have announced a 3rd indication, spinal muscular atrophy (SMA). We believe ARGX-119 has potential as a novel treatment modality in multiple serious indications.
Our immunology innovation program (IIP) is a key driver for our future sustainable growth. This is reflected in 4 new investigational new drugs (INDs) that will start Phase 1 clinical trials in 2025 to continue to deliver immunology innovations to the patients who need them.
The science of co-creation drives our quest to engineer life-changing immunology solutions, the resilient spirit of patients fuels our urgency to deliver them. The infinity sign symbolizes our commitment to science and patients; it has no bounds. Our potential is infinite. Our purpose is immunology innovation.
VYVGART
Reach More Patients Globally with VYVGART
- VYVGART is now approved in the U.S., Japan, the EU, the UK, Switzerland, Israel, Mainland China, Canada, South Korea, United Arab Emirates, Australia and Kuwait (through Genpharm Services FZ-LLC (Genpharm)) for the treatment of gMG. VYVGART is now also approved and launched in Japan for the treatment of ITP. VYVGART SC is now approved in the U.S., the EU, the UK, Japan, China (through Zai Lab), Australia and Kuwait (through Genpharm) for the treatment of gMG and in the U.S., Japan, and China for the treatment of CIDP. VYVGART is the only gMG treatment available as both an intravenous (IV) and a simple SC injection, providing choice to patients in how and where they are treated.
- In 2024, we generated product net sales of $2.2 billion.
- Pricing and reimbursement discussions for VYVGART and VYVGART SC remain ongoing in multiple jurisdictions, including in several countries in the EU, with new agreements in place in France, Luxembourg, Belgium, the Netherlands, Poland, Slovakia and Austria.
- We filed for approval of VYVGART for gMG in Saudi Arabia and are expecting a decision on approval in 2025.
- We filed for approval of VYVGART SC for CIDP in the EU and are expecting a decision on approval in 2025.
- We received approval of the PFS for gMG in Europe on February 13, 2025.
- We filed for approval of the PFS for gMG and CIDP in the U.S. with a PDUFA Date of April 10, 2025. We also filed for approval in Canada and Japan with expected decisions on approval in 2025.
Advance Extensive Pipeline
We continue to demonstrate breadth and depth within our immunology pipeline and have advanced multiple pipeline-in-a-product candidates. With efgartigimod, we are furthering our leadership in neonatal Fc receptor (FcRn) and we are continuing its development in more than 10 indications today. Beyond efgartigimod, we are advancing our other clinical pipeline programs, including empasiprubart (C2 inhibitor) which has now shown proof-of-concept in MMN and initiated its first Phase 3 clinical trial, and is in Phase 2 POC clinical trials in delayed graft function (DGF) and DM. We also announced CIDP as the 4th indication for empasiprubart during our R&D Day on July 16, 2024 and plan to start a registrational study in CIDP evaluating empasiprubart head-to-head versus intravenous IgG (IVIg) in first half of 2025.
In addition, we have initiated Phase 1b/2a clinical trials of ARGX-119, a MuSK agonist, in CMS congenital myasthenic syndromes and ALS. Four new pipeline candidates were nominated in 2024 from our immunology innovation program (IIP), including: ARGX-213, ARGX-121 and ARGX-220 and ARGX-109. Phase 1 results expected for ARGX-109 in second half of 2025 and for ARGX-213 and ARGX-121 in first half of 2026.
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Build out the Innovation Ecosystem
In January 2024, we announced the nomination of four new pipeline candidates, including: ARGX-213 targeting FcRn, furthering argenx’s leadership in this new class of medicine; ARGX-121 targeting Immunoglobulin A (IgA) and ARGX-220, which are first-in-class targets broadening argenx’s focus across the immune system; and ARGX-109, targeting IL-6, which plays an important role in inflammation. Preclinical work is ongoing in each candidate and the first healthy volunteer studies are expected to start in 2025.
See Our Products and Product CandidatesNeurology indications:
- CIDP (ADHERE): following the positive topline results from the ADHERE clinical trial in CIDP, a supplemental biologics license application (sBLA) for efgartigimod SC was approved for the treatment of CIDP and launched in July 2024 in the U.S. We also received approval in Japan and China for the treatment of CIDP.
- Myositis (ALKIVIA): In November 2024, we announced the GO decision to continue Phase 3 of the ALKIVIA clinical trial.
- The decision to continue clinical development of efgartigimod SC in each of the three myositis subtypes, including IMNM, ASyS and DM, is supported by the efficacy and safety results from the Phase 2 portion of the seamless Phase 2/3 ALKIVIA clinical trial. Overall, the clinical trial met its primary endpoint, demonstrating a statistically significant treatment effect in mean total improvement score (TIS) at week 24, and showed improvement across all six core set measures of the TIS in favor of efgartigimod SC compared to placebo. The observed safety and tolerability profile was consistent to that demonstrated with other clinical trials.
- Topline results expected 2H 2026.
- TED (UplighTED): registrational clinical trials in thyroid eye disease (TED) ongoing with efgartigimod PFS. Topline results expected 2H 2026.
- Seronegative gMG (ADAPT-SERON): registrational clinical trial in seronegative gMG patients ongoing with efgartigimod IV. Topline results expected 2H 2025.
- Ocular MG (ADAPT-OCULUS): registrational clinical trial in ocular MG patients ongoing with efgartigimod PFS. Topline results expected 1H 2026.
Hematology/rheumatology indications:
- ITP (ADVANCE-IV): positive clinical trial results formed the basis of approval in Japan for ITP, received on March 26, 2024.
- ITP (ADVANCE-NXT): confirmatory clinical trial in ITP ongoing with efgartigimod IV in the U.S. Topline results expected in 2H 2026.
- Primary SjD (RHO): following the analysis of topline data from the Phase 2 POC clinical trial through our partnership with IQVIA Ltd (IQVIA) in SjD we decided to continue the development of efgartigimod PFS to Phase 3 (UNITY), which was initiated at the end of 2024. Topline results expected in 2027.
- Systemic Sclerosis (SSc): Phase 2 POC clinical trial ongoing. Topline results expected in 2H 2026.
Nephrology indications:
- Lupus Nephritis: Phase 2 POC clinical trial ongoing through our partnership with Zai Lab. Topline results expected in 2H 2025.
- Antibody-mediated rejection: shAMRock Phase 2 POC clinical trial in antibody mediated rejection (AMR) has been initiated.
Other indications:
- In 2024 we made the decision to stop development in PC-POTS (ALPHA), Bullous Pemphigoid (BALLAD) and membranous nephrology based on review of the Phase 2 data.
Corporate Achievements
Dr. Brian Kotzin
Dr. Brian Kotzin joined the Board of Directors in May 2024 as a non-executive director and chairperson of the research and development committee
See Board of DirectorsMr. Peter Verhaeghe
Mr. Peter Verhaeghe, who has served as a non-executive director since July 2014, was reappointed as a non-executive director and chairperson of the Board of Directors for a term of 2 years
See Board of DirectorsDr. Pamela Klein
Dr. Pamela Klein, who has served as a non-executive director since April 2016, was reappointed as a non-executive director for a term of 2 years
See Board of Directors1,599
Employees
Expansion to 1,599 full-time employees (as of December 31, 2024) to support further growth of our business, including fully staffed commercial teams in the U.S., Europe, Japan and Canada
See Employees
Financial Highlights
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Product net sales
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Research & development
Transition to sustainable operating profitability in 2025 enables continued investment in innovation.
See our Financial Review