RegulationPriority Review (United States), Fast Track (United States), Orphan Drug (United States), Orphan Drug (European Union), Breakthrough Therapy (China), Orphan Drug (South Korea), Paediatric investigation plan (European Union), Promising Innovative Medicine (United Kingdom), Priority Review (China)

Structure/Sequence

Sequence Code 18457995

Source: *****

Clinical Trials associated with Efgartigimod Alfa

NCT07011589

/ Not yet recruitingPhase 1/2IIT

Targeting Collagen VII Antibodies in Bullous Diseases Using Efgartigimod IV (VYVGART)

The study objective is to see if IV Efgartigimod and Vyjuvek treatment in Recessive Dystrophic Epidermolysis Bullosa (RDEB) and IV Efgartigimod treatment in Epidermolysis Bullosa Acquisita (EBA) improves wound healing and affects the levels of C7 antibody levels in serum.
Fewer wounds, more rapidly healing wounds, and decreased C7 antibodies could improve quality of life.

Start Date01 Jul 2025

Sponsor / Collaborator

Stanford University

[+1]

NCT06831058

/ RecruitingPhase 2IIT

A Pilot Study of Efgartigimod for Immune-mediated Thrombotic Thrombocytopenic Purpura (iTTP)

Immune-mediated Thrombotic thrombocytopenic purpura (iTTP) is a rare, autoimmune disorder characterized by life-threatening episodes of thrombocytopenia, microangiopathic hemolytic anemia and organ damage. Patients have an unpredictable course punctuated by relapses associated with autoantibody-mediated (primarily IgG) depletion of ADAMTS13, a key regulator of coagulation. ADAMTS13 deficiency during remission has been associated with increased risk of relapse, but also, and potentially more devastating, ischemic stroke.
Until recently, it was presumed that rituximab (a monoclonal antibody targeting B cells) improved relapse-free survival in most patients, but this was based on findings from very small studies. Given concern about stroke and relapse risk, preventive immunosuppression with rituximab has also recently come into practice for patients with falling ADAMTS13 activity (ADAMTS13-relapse). It is expected that following efgartigimod therapy, there will be a rise in ADAMTS13 activity to the normal range that will be sustained during the treatment period. Following withdrawal of therapy, it is expected that most participants will experience a fall in ADAMTS13 activity, demonstrating the safety and efficacy in efgartigimod to reliably but temporarily reduce pathogenic antibodies. This would demonstrate the potential efficacy for efgartigimod as a maintenance therapy to safely prevent relapse of iTTP to be further explored in a larger efficacy study.

Start Date01 May 2025

Sponsor / Collaborator

University of Minnesota

NCT06909214

/ RecruitingPhase 4

A Phase 4, Open-Label, Prospective, Single-Group, Multicenter Study to Evaluate the Clinical Outcomes of Efgartigimod PH20 SC in Adult Participants With New-Onset Generalized Myasthenia Gravis

The main purpose of this study is to measure how well adults with new-onset gMG (which means they've had generalized disease signs and/or symptoms for less than 1 year) respond to treatment with efgartigimod PH20 SC. The study consists of a treatment period of 51 weeks. The study duration for each participant will be approximately 58 weeks.

Start Date17 Apr 2025

Sponsor / Collaborator

argenx SE

100 Clinical Results associated with Efgartigimod Alfa

100 Translational Medicine associated with Efgartigimod Alfa

100 Patents (Medical) associated with Efgartigimod Alfa

193

Literatures (Medical) associated with Efgartigimod Alfa

31 Dec 2025·Human Vaccines & Immunotherapeutics

Impact of FcRn antagonism on vaccine-induced protective immune responses against viral challenge in COVID-19 and influenza mouse vaccination models

Article

Author: Vanhoenacker, Peter ; Baumeister, Judith ; Wouters, Heidi ; Schotsaert, Michael ; Laghlali, Gabriel ; Ulrichts, Peter ; Chang, Lauren A ; Singh, Gagandeep ; Binazon, Ornella ; Warang, Prajakta ; Elhemdaoui, Nadia ; Augustynková, Kateřina ; Moshir, Mahan ; Steeland, Sophie ; Notebaert, Margo

Antagonism of the neonatal Fc receptor through an engineered antibody Fc fragment, such as efgartigimod, results in a decrease in immunoglobulin G levels. This approach is being evaluated as a therapeutic strategy for the treatment of IgG-mediated autoimmune diseases. Our goal was to evaluate the impact of mFc-ABDEG, a mouse-adapted antibody Fc fragment with a mode of action highly similar to efgartigimod, on vaccine-induced protective immune responses against viral infections. Therefore, mouse vaccination models for COVID-19 and influenza were employed, utilizing an mRNA COVID-19 vaccine (COMIRNATY) and an adjuvanted, inactivated quadrivalent influenza vaccine (Seqirus+AddaVax), respectively. In both models, vaccination induced robust humoral responses. As expected, animals treated with mFc-ABDEG had lower levels of virus-specific IgG, while virus-specific IgM responses remained unaffected. The COVID-19 vaccine induced a strong Th1-type T cell response irrespective of mFc-ABDEG treatment. Influenza vaccination resulted in a poor T cell induction, regardless of mFc-ABDEG treatment, due to the Th2-biased response that inactivated influenza vaccines typically induce. Importantly, mFc-ABDEG treatment had no effect on protective immunity against live viral challenges in both models. Vaccinated animals treated with mFc-ABDEG were equally protected as the non-treated vaccinated controls. These non-clinical data demonstrate that FcRn antagonism with mFc-ABDEG did not affect the generation of vaccine-induced protective humoral and cellular responses, or protection against viral challenges. These data substantiate the clinical observations that, although IgG titers were reduced, FcRn antagonism with efgartigimod did not impair the ability to generate new specific IgG responses, regardless of the timing of vaccination.

01 Jul 2025·CLINICAL NEUROLOGY AND NEUROSURGERY

Efficacy and safety of FcRn inhibitors in patients with Myasthenia gravis: An updated systematic review and meta‑analysis

Review

Author: Hanif, Ummara ; Muzammil, Anam ; Farooqi, Hanzala Ahmed ; Hassan, Syed Muhammad ; Akhtar, Muzamil ; Maryam, Ayesha ; Khan, Zarak ; Athar, Zoya ; Akhtar, Mehmood

BACKGROUND:

Myasthenia gravis (MG) is a chronic, complex autoimmune disorder characterized by the production of autoantibodies that destroy neuromuscular junctions. Blocking the neonatal Fc receptors (FcRn) enhances IgG catabolism, offering a novel therapeutic approach.

METHODS:

PubMed, Embase, and the Cochrane Library were searched up to February 2025, for RCTs evaluating FcRn inhibitors in MG. A random effects model to calculate pooled risk ratios (RR) and mean differences with 95 % confidence intervals (CI).

RESULTS:

873 patients from 8 randomized control trials (RCTs) were analyzed. Compared to placebo, FcRn inhibitors significantly reduced Myasthenia Gravis Activities of Daily Living (MG-ADL) scores (MD of -1.45 [95 % CI, -1.91 to -0.99]; P < 0.00001), Quantitative Myasthenia Gravis( QMG) scores (MD = -2.33 [95 % CI, -3.57 to -1.09]; P = 0.0002), and Myasthenia Gravis Composite (MGC) scores (MD = -2.96 [95 % CI, -4.29 to -1.63]; P < 0.0001). The FcRn inhibitors improved MG-ADL responder rates (RR = 1.60 [95 % CI, 1.27-2.02]; P < 0.0001), and Myasthenia Gravis Quality of Life (MGQoL15r) scores (MD = -2.18 [95 % CI, -3.35 to -1.00]; P = 0.0003). Serious adverse events were lower with the FcRn inhibitors (32/519) than the placebo (39/397). Subgroup analysis revealed that Rozanolixizumab and Nipocalimab improved MG-ADL scores, but had inferior responder rates. Additionally, Rozanolixizumab significantly improved MGC scores but had more adverse events.

CONCLUSION:

FcRn inhibitors demonstrated good efficacy and safety in MG, with efgartigimod and nipocalimab showing strong efficacy without added risk. Further research is required to evaluate long-term outcomes and optimize treatment.

01 Jun 2025·MUSCLE & NERVE

Sustained Minimal Manifestations With Extended Interval Dosing of Efgartigimod in Seronegative Myasthenia Gravis: Case Report

Letter

Author: Iguchi, Yasuyuki ; Matsuno, Hiromasa ; Komatsu, Teppei ; Nakayama, Yoshitaka ; Motegi, Haruhiko ; Mitsumura, Hidetaka

307

News (Medical) associated with Efgartigimod Alfa

11 Jun 2025

·firstwordpharma.com

Argenx's FcRn inhibitor Vyvgart hits targets in Phase II myositis, Sjögren's studies

Argenx unveiled encouraging mid-stage data for Vyvgart (efgartigimod alfa) in myositis and Sjögren's disease, adding two more autoimmune diseases to the list of conditions that the FcRn inhibitor has shown promise in. The results were presented Wednesday at the European Congress of Rheumatology (EULAR).Vyvgart — which reduces pathogenic IgG by blocking FcRn-mediated recycling — is currently approved to treat generalised myasthenia gravis (gMG) and chronic inflammatory demyelinating polyneuropathy. "The accumulating body of evidence about the role of IgG autoantibodies reinforces the therapeutic potential of efgartigimod…for treating several rheumatic diseases — aiming to go beyond symptom management by targeting the underlying disease," said Luc Truyen, chief medical officer of argenx.Goals met in myositisThe ongoing 240-participant Phase II/III ALKIVIA study is investigating subcutaneous Vyvgart across three subtypes of idiopathic inflammatory myopathies, with the Phase II portion involving 90 patients.Results showed that Vyvgart met the primary endpoint, demonstrating a statistically significant mean Total Improvement Score (TIS) of 50.45 compared with 35.65 for placebo at 24 weeks. Moreover, clinical response rates favoured the study drug: 79% of Vyvgart-treated patients achieved moderate improvement (TIS ≥40) versus 47% receiving placebo, while 34% demonstrated major improvement (TIS ≥60) compared with 9.5% in the control arm. For one of the secondary endpoints, Vyvgart led to faster improvement than placebo, with a median time to TIS ≥20 of 30 days versus 72 days for placebo and TIS ≥40 in 16 weeks, which most placebo recipients did not achieve. In terms of safety, the therapy was well tolerated, with similar treatment-emergent adverse event rates between groups. The drug continues to be evaluated in ALKIVIA’s Phase III portion.Sjögren's study follows suitMeanwhile, the Phase II RHO study randomised 34 patients with Sjögren's disease to receive Vyvgart or placebo for up to 24 weeks. The primary endpoint was CRESS, a composite of five measures: systemic activity, patient-reported outcomes, tear and salivary gland function and serology.Data showed the trial met its primary goal, with 45.5% of Vyvgart recipients achieving improved outcomes on the CRESS measure at week 24, compared with 11.1% receiving placebo. A key secondary endpoint, the cSTAR composite of five disease measures, also showed a 54.5% response with Vyvgart versus 33.3% with placebo.Additionally, biomarker analysis revealed that Vyvgart led to sustained IgG reduction of nearly 60% from week four onwards, with notable decreases in anti-Ro52, rheumatoid factor and C1Q immune complexes compared with placebo. Vyvgart also exhibited a favourable safety profile, consistent with prior trials and without new safety signals. The ongoing Phase III UNITY trial is further evaluating its efficacy and safety in moderate to severe disease.The company is investigating Vyvgart in over 15 severe autoimmune diseases driven by pathogenic IgG antibodies.

Clinical ResultPhase 2Phase 3Drug Approval

09 Jun 2025

·www.einpresswire.com

Beyond the Rash: Emerging Innovation Fuels a New Era in Dermatomyositis Therapy | Competitive Intelligence

Novel biologics & small molecules aim to disrupt dermatomyositis treatment, with late-stage pipeline therapies promising precision, and better outcomes. Dermatomyositis drug development is entering a new era, where targeted immunology meets real-world need-this space is no longer stagnant, it's becoming strategically vibrant.” — DataM Intelligence AUSTIN, TX, UNITED STATES, June 9, 2025 / EINPresswire.com / -- Dermatomyositis (DM), a chronic autoimmune condition impacting both muscle and skin, has historically stood at the crossroads of clinical ambiguity and limited therapeutic options. Marked by progressive muscle weakness, characteristic skin rashes, and systemic manifestations such as lung or cardiac involvement, DM not only challenges diagnostic clarity but also demands long-term and nuanced disease management. Yet despite the complexity of its pathology, the therapeutic armamentarium has remained surprisingly narrow-until now. Recent scientific momentum and increasing disease awareness are triggering a paradigm shift in how the medical community approaches dermatomyositis. From novel immunomodulatory mechanisms to patient-friendly delivery formats, a wave of late-stage pipeline candidates is positioning itself to redefine treatment outcomes and patient experience. Book Your Free CI Consultation Call: https://www.datamintelligence.com/strategic-insights/ci/dermatomyositis Understanding the Landscape: A Rare Disease with High Unmet Needs Dermatomyositis is rare, with an estimated incidence of 1.4 cases and prevalence of 5.8 cases per 100,000 individuals in the United States. It disproportionately affects females and tends to emerge more prominently in middle-aged to older adults. Although the exact etiology remains unclear, evidence points toward a multifactorial immune-mediated process potentially triggered by viral infections, certain medications, or even underlying malignancies. From a treatment standpoint, the landscape has been frustratingly limited. Until 2021, no therapies had gained FDA approval specifically for dermatomyositis. Immunosuppressants and corticosteroids have been mainstays, though often with significant toxicity, variable efficacy, and a lack of long-term disease control. That changed with the approval of Octagam 10%, an intravenous immunoglobulin (IVIG) product, which brought a long-awaited formal option to the table. Octagam 10%: The First and Only FDA-Approved Option Octagam 10%, developed by Octapharma and co-marketed with Pfizer, gained FDA approval in July 2021 for adult patients with dermatomyositis. It works by delivering pooled human IgG antibodies that modulate immune responses. While Octagam offers benefit, especially for patients unresponsive to conventional treatments, its use comes with certain limitations: - Administration: Requires intravenous infusion in a clinical setting every 3–4 weeks, which is logistically challenging for many patients. - Efficacy: While beneficial in refractory cases, it does not significantly modify the underlying disease or offer long-term remission. - Safety: Known risks include thromboembolic events, hemolysis, and infusion-related complications. Despite these issues, Octagam has established itself as the current standard of care. However, the treatment paradigm is on the cusp of disruption. The Emerging Pipeline: Precision Immunology at the Helm The dermatomyositis drug development pipeline is now flush with innovation. Companies are advancing targeted therapies that not only offer improved efficacy but also focus on optimizing the patient experience. Among the most promising candidates are: - Anifrolumab (AstraZeneca): A subcutaneously administered monoclonal antibody targeting the Type I interferon receptor (IFNAR1). Currently in Phase III trials, Anifrolumab offers potential advantages in targeting a key immunologic driver while eliminating the need for intravenous infusions. - Dazukibart (Pfizer): An intravenous monoclonal antibody targeting interferon-beta (IFNB1). As a competitor to Octagam, it aims to provide more precise suppression of inflammatory cytokines involved in disease pathology. - Efgartigimod SC (argenx): A subcutaneous anti-FcRn monoclonal antibody in Phase III trials. It works by reducing pathogenic IgG levels and provides a more convenient route of administration. - Brepocitinib (Priovant Therapeutics): An oral small molecule dual TYK2/JAK1 inhibitor currently in Phase III. It combines efficacy with ease-of-use, potentially reducing reliance on systemic steroids. - Rapcabtagene autoleucel (Novartis): A CD19-directed CAR-T cell therapy in Phase II development. While still early, this cell-based approach may provide a breakthrough option for severe or treatment-resistant cases. - Daxdilimab (Amgen) and Empasiprubart (argenx): Novel monoclonal antibodies targeting ILT7 and C2 complement protein, respectively, and both are in Phase II. - Enpatoran (Merck KGaA): An oral TLR7/8 antagonist aiming to calm innate immune system overactivation-another promising option in mid-stage development. Strategic Trends and Competitive Insights What sets these therapies apart isn’t just novel mechanisms of action-it’s their alignment with strategic clinical trends: Convenience and Compliance: The burden of monthly IV infusions is driving a shift toward oral and subcutaneous (SC) delivery formats, enabling at-home administration and improved adherence. Targeted Modulation vs Broad Suppression: Unlike traditional immunosuppressants that act broadly, emerging therapies aim for precise immunologic targets such as interferons, FcRn, JAK/STAT, and complement systems-offering better risk-benefit profiles. Oral Innovation Rising: Agents like Brepocitinib and Enpatoran show that small molecules still have a significant place in the immunology pipeline-offering scalable, patient-friendly treatment options without biologic infusion overhead. Refractory & Rare Subsets: Treatments like Rapcabtagene autoleucel could cater to patients with refractory disease or unique subtypes like juvenile dermatomyositis, where conventional therapies fall short. The Target Opportunity Profile: What New Drugs Must Beat To challenge Octagam’s dominance, pipeline drugs must surpass a well-established benchmark. Here's how the target opportunity profile breaks down: - Efficacy: Must offer equal or superior impact on muscle strength, skin symptoms, and overall disease activity-preferably with faster onset and steroid-sparing benefits. - Safety: Lower risk of infusion-related complications, thrombotic events, and immunogenicity will give new entrants a competitive edge. - Convenience: Oral or SC administration with less frequent dosing could transform patient quality of life. - Innovation: First-in-class mechanisms like ILT7 inhibition, FcRn blockade, or JAK1/TYK2 dual inhibition represent true clinical advancement. If these parameters are met-or even partially exceeded-Octagam’s market stronghold may rapidly erode. Download Free CI Sample Report: https://www.datamintelligence.com/strategic-insights/sample/dermatomyositis The Road Ahead: A Time of Strategic Transition Dermatomyositis is no longer a stagnant field. Driven by deeper immunological understanding and patient-centered innovation, the current pipeline suggests that a true therapeutic evolution is underway. Whether through small molecules, biologics, or even cell therapies, the next generation of treatments aims to offer not just symptom management but real disease control. As regulatory milestones approach in the coming months and years, stakeholders across the healthcare continuum-from clinicians to patients to payers-must prepare for a reshaped treatment landscape. Read Related CI Reports: 1. Autosomal Dominant Polycystic Kidney Disease 2. Prurigo Nodularis | Competitive Intelligence Sai Kumar DataM Intelligence 4market Research LLP +1 877-441-4866 email us here Visit us on social media: LinkedIn X Legal Disclaimer: EIN Presswire provides this news content "as is" without warranty of any kind. We do not accept any responsibility or liability for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this article. If you have any complaints or copyright issues related to this article, kindly contact the author above.

ImmunotherapyPhase 3Drug ApprovalPhase 2Cell Therapy

22 May 2025

·www.prnewswire.com

Johnson & Johnson's IMAAVY Gets FDA Approval, Taking on Top Players in Myasthenia Gravis Market | DelveInsight

The FDA has approved Johnson & Johnson's anti-FcRn antibody, nipocalimab, for the treatment of generalized myasthenia gravis. The drug will be sold under the brand name IMAAVY. This approval positions J&J to challenge leading competitors in the myasthenia gravis market, such as AstraZeneca, which markets the monoclonal antibodies SOLIRIS and ULTOMIRIS. J&J will also be competing with Argenx's VYVGART and UCB's RYSTIGGO and ZILBRYSQ. LAS VEGAS, May 22, 2025 /PRNewswire/ -- Myasthenia gravis is an autoimmune disorder in which antibodies disrupt the communication between nerves and muscles, resulting in skeletal muscle weakness. This weakness primarily affects voluntary muscles that control eye movement, facial expression, swallowing, and limb function. The condition is predominantly driven by autoantibodies targeting either the acetylcholine receptor (AChR-Ab) or a receptor-associated protein known as muscle-specific tyrosine kinase (MuSK-Ab). According to DelveInsight's analysis, there were approximately 300,000 diagnosed prevalent cases of myasthenia gravis across the 7MM in 2024. This number is expected to grow steadily over the forecast period (2025–2034), with a projected CAGR. The current myasthenia gravis therapeutics market mainly consists of symptomatic treatments such as acetylcholinesterase inhibitors like Mestinon. However, achieving full clinical remission with symptomatic therapy alone is challenging. As a result, immunosuppressive drugs are frequently introduced. First-line immunotherapy typically involves high-dose oral corticosteroids such as prednisone or prednisolone, which can induce a rapid response within 2 to 4 weeks. To reduce reliance on steroids and improve long-term disease control, nonsteroidal immunosuppressants (NSISTs) are often used. These include antimetabolites (e.g., azathioprine, mycophenolate mofetil, methotrexate, and cyclophosphamide) and calcineurin inhibitors (e.g., tacrolimus and cyclosporine). While NSISTs may be used as standalone treatments, they usually take 6 to 9 months to produce noticeable effects. Immunomodulatory therapies, including intravenous immunoglobulin (IVIg) and plasmapheresis (PLEX), are typically reserved for acute exacerbations or myasthenic crises. They are also employed as chronic treatments in patients who cannot tolerate or do not respond to standard immunosuppressants. Although traditional immunosuppressive strategies can help manage symptoms, achieving long-term, stable remission remains difficult. Additionally, about 15% of patients are considered treatment-refractory, meaning they either fail to respond to or cannot tolerate multiple immunosuppressive options. These patients often face the dual challenge of persistent symptoms and the adverse effects of long-term immunosuppression. Learn more about the myasthenia gravis disease market @ Myasthenia Gravis Treatment Medications The myasthenia gravis treatment market is well-established, with multiple approved drugs for myasthenia gravis, including SOLIRIS, ULTOMIRIS, RYSTIGGO, ZILBRYSQ, and VYVGART, among others. Alexion Pharmaceuticals' SOLIRIS is a monoclonal antibody that targets and inhibits complement protein C5, thereby blocking the formation of the terminal complement complex. This action helps reduce complement-driven hemolysis in PNH and thrombotic microangiopathy in aHUS. Although its exact mechanism in gMG and NMOSD is not fully known, it is believed to decrease complement complex accumulation. Between 2017 and 2023, SOLIRIS gained regulatory approvals in Japan, the US, and the EU for treating gMG, including pediatric indications in Japan. ULTOMIRIS (ravulizumab), also from Alexion Pharmaceuticals, is the first long-acting C5 inhibitor, offering rapid, complete, and sustained suppression of complement activity. It binds to C5, preventing its cleavage and subsequent formation of the membrane attack complex (C5b-9). ULTOMIRIS is effective in managing terminal complement-mediated hemolysis in PNH and thrombotic microangiopathy in aHUS. Although the precise mechanism in gMG and NMOSD remains unclear, it is thought to reduce complement deposition at the neuromuscular junction and block aquaporin-4 antibody-mediated complement activation. It received approvals from the US FDA, EMA, and Japan's PMDA in 2022. Get an in-depth assessment on generalized myasthenia gravis medication for adults @ ULTOMIRIS vs SOLIRIS for Myasthenia Gravis UCB Biopharma's RYSTIGGO is a subcutaneously administered, humanized monoclonal antibody that binds with high affinity to the human neonatal Fc receptor (FcRn). It received regulatory approval from the US FDA and Japan's PMDA in 2023, followed by the EC in January 2024. ZILBRYSQ, also developed by UCB Biopharma, is the first once-daily subcutaneous peptide-based inhibitor specifically targeting complement protein C5. It works by blocking C5 cleavage and the formation of the terminal complement complex C5b-9. Although its precise action in gMG remains uncertain, it is thought to limit C5b-9 accumulation at the neuromuscular junction. ZILBRYSQ gained approval from the US FDA, EMA, and PMDA in 2023. VYVGART, from Argenx, is a human IgG1 antibody fragment that targets FcRn, leading to a decrease in circulating IgG autoantibodies. It is the first FcRn blocker to receive regulatory approval. Argenx's VYVGART HYTRULO (also marketed as VYVDURA) combines efgartigimod alfa, an FcRn inhibitor, with hyaluronidase, an endoglycosidase, for treating adults with generalized myasthenia gravis. This formulation was approved by the US FDA and EMA in 2023 and by the PMDA in January 2024. Uncover the mechanism of FcRn inhibitor myasthenia gravis medication @ Myasthenia Gravis MoA On April 30, 2025, the FDA approved Johnson & Johnson's nipocalimab, branded as IMAAVY, as a new therapy for gMG. This approval puts IMAAVY among the list of approved drugs for myasthenia gravis treatment. The approval, which J&J says encompasses the widest eligible patient population with gMG, includes individuals aged 12 and above who test positive for AChR or anti-muscle-specific kinase (MuSK) antibodies. IMAAVY functions by inhibiting the FcRn protein, which plays a role in maintaining circulating IgG antibodies, key drivers in many autoimmune disorders. By blocking FcRn, IMAAVY significantly reduces harmful IgG autoantibodies while preserving the body's overall immune response, according to clinical data. J&J sees broader potential for IMAAVY beyond gMG, believing its mechanism of action could apply across multiple autoimmune conditions due to IgG's involvement in various diseases. The FDA's decision followed results from the ongoing Phase III Vivacity-MG3 trial, which evaluates IMAAVY in combination with standard care versus placebo and standard care in 199 adult gMG patients, including 153 antibody-positive individuals. Additionally, J&J is conducting a Phase II/III pediatric trial called Vibrance, where IMAAVY, combined with standard care, achieved its primary goal by reducing total serum IgG levels by 69% over 24 weeks. J&J acquired IMAAVY through its USD 6.5 billion acquisition of Momenta Pharmaceuticals in 2020, with the FcRn inhibitor myasthenia gravis serving as the centerpiece of that deal. The company is aiming for blockbuster status, forecasting peak annual sales of over USD 5 billion for the myasthenia gravis medication. Dive deep into the myasthenia gravis treatment cost @ Generalized Myasthenia Gravis Drug with Copay Myasthenia gravis pipeline possesses some drugs in mid- and late-stage development to be approved in the near future. The expected launch of myasthenia gravis treatment medications, such as Batoclimab (Immunovant Sciences GmbH), Descartes-08 (Cartesian Therapeutics), and others, are expected to further create a positive impact on the myasthenia gravis disease market. Discover which therapies are expected to grab major myasthenia gravis therapeutics market share @ Market Analysis on Myasthenia Gravis Batoclimab is a fully humanized monoclonal antibody designed to inhibit neonatal Fc receptors by blocking the interaction between FcRn and IgG, thereby promoting the breakdown of autoantibodies. It has received Orphan Drug Designation (ODD) from both the US FDA and the EMA. In March 2025, Immunovant announced topline results from its Phase III trial evaluating batoclimab in patients with myasthenia gravis. Descartes-08 represents the first RNA-engineered CAR T-cell (rCAR-T) therapy developed for autoimmune conditions. This therapy uses cytotoxic T-cells programmed to target and eliminate pathogenic plasma cells that produce autoantibodies. Created from a patient's own blood, Descartes-08 has also received ODD from the US FDA for treating myasthenia gravis. In December 2024, Cartesian Therapeutics shared encouraging updated findings from its Phase IIb study of Descartes-08 in myasthenia gravis. Furthermore, in January 2025, the FDA agreed to the company's Phase III AURORA trial design under a Special Protocol Assessment, with trial initiation planned for the first half of 2025. Curious about what the new myasthenia gravis treatments are in development? Visit Myasthenia Gravis Clinical Trials The anticipated launch of these emerging myasthenia gravis treatments are poised to transform the market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the myasthenia gravis market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth. DelveInsight estimates that the myasthenia gravis market size in the 7MM is expected to grow from USD 5.2 billion in 2024 at a significant CAGR by 2034. According to DelveInsight's analysis, the myasthenia gravis market growth is anticipated with the introduction of emerging therapies, leading to an expansion in the myasthenia gravis market size throughout the forecast period (2025–2034). This anticipated myasthenia gravis market growth is driven by advancements in treatment options, greater healthcare access, and a rising myasthenia gravis prevalence, which together foster higher demand for innovative and effective therapies. DelveInsight's latest published market report titled Myasthenia Gravis Market Insight, Epidemiology, and Market Forecast – 2034 will help you to discover which myasthenia gravis treatment market leader is going to capture the largest market share. The report provides comprehensive insights into the country-specific myasthenia gravis treatment guidelines, patient pool analysis, and epidemiology forecast to help understand the key opportunities and assess the market's underlying potential. The myasthenia gravis market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM, segmented into: Total Diagnosed Prevalent Cases of Myasthenia Gravis Gender-specific Diagnosed Prevalent Cases of Myasthenia Gravis Age-specific Diagnosed Prevalent Cases of Myasthenia Gravis Diagnosed Prevalent Cases of Myasthenia Gravis by MGFA Classification Diagnosed Prevalent Cases of Generalized Myasthenia Gravis by Antibody Serology The report provides an edge while developing business strategies by understanding trends shaping and driving the 7MM myasthenia gravis market. Highlights include: 10-year Forecast 7MM Analysis Epidemiology-based Market Forecasting Historical and Forecasted Market Analysis upto 2034 Emerging Drug Market Uptake Peak Sales Analysis Key Cross Competition Analysis Industry Expert's Opinion Access and Reimbursement Download this myasthenia gravis market report to assess the epidemiology forecasts, understand the patient journeys, know KOLs' opinions about the upcoming treatment paradigms, and determine the factors contributing to the shift in the myasthenia gravis market. Also, stay abreast of the mitigating factors to improve your market position in the myasthenia gravis therapeutic space. Related Reports Myasthenia Gravis Epidemiology Forecast Myasthenia Gravis Epidemiology Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted myasthenia gravis epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan. Myasthenia Gravis Pipeline Myasthenia Gravis Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key myasthenia gravis companies, including Harbour BioMed (Guangzhou) Co. Ltd., Kyverna Therapeutics, Cabaletta Bio, Takeda, Hoffmann-La Roche, Immunovant Sciences GmbH, Regeneron Pharmaceuticals, Novartis Pharmaceuticals, Janssen Research & Development, LLC, Momenta Pharmaceuticals, Inc., Amgen, Dianthus Therapeutics, Cartesian Therapeutics, COUR Pharmaceutical Development Company, Inc., Alexion Pharmaceuticals, Inc., among others. Generalized Myasthenia Gravis Market Generalized Myasthenia Gravis Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key gMG companies including Alexion Pharmaceuticals, Argenx, UCB Biopharma, Argenx-Halozyme Therapeutics, Horizon Therapeutics, Hoffmann-La Roche, Janssen Research & Development, LLC, Immunovant Sciences GmbH, Bristol Myers Squibb, Biogen Inc., Pfizer Inc., Novartis AG, Sanofi S.A., Roche Holding AG, Johnson & Johnson, Takeda Pharmaceutical Company Limited, AbbVie Inc., Eli Lilly and Company, Merck & Co., Inc., GlaxoSmithKline plc, AstraZeneca plc, Amgen Inc., Boehringer Ingelheim International GmbH, Genentech, Inc., Gilead Sciences, Inc., Celgene Corporation, Vertex Pharmaceuticals Incorporated , among others. Generalized Myasthenia Gravis Pipeline Generalized Myasthenia Gravis Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key gMG companies, including Biocon, Cartesian Therapeutics, UCB, Momenta Pharmaceuticals, HanAll Biopharma, Roche, Alexion, Novartis, Takeda, BioMarin, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

ImmunotherapyPhase 3Drug ApprovalOrphan DrugClinical Result

100 Deals associated with Efgartigimod Alfa

External Link

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-	Efgartigimod Alfa	-	DB15270

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Purpura, Thrombocytopenic, Idiopathic	Japan	argenx SE	26 Mar 2024
Myasthenia Gravis	United States	argenx BV	17 Dec 2021

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Polyradiculoneuropathy, Chronic Inflammatory Demyelinating	NDA/BLA	China	Zai Lab (Shanghai) Co., Ltd.	24 Apr 2024
Primary Sjögren's syndrome	Phase 3	United States	argenx SE	15 Jan 2025
Primary Sjögren's syndrome	Phase 3	Japan	argenx SE	15 Jan 2025
Primary Sjögren's syndrome	Phase 3	Austria	argenx SE	15 Jan 2025
Primary Sjögren's syndrome	Phase 3	Belgium	argenx SE	15 Jan 2025
Primary Sjögren's syndrome	Phase 3	Bulgaria	argenx SE	15 Jan 2025
Primary Sjögren's syndrome	Phase 3	Canada	argenx SE	15 Jan 2025
Primary Sjögren's syndrome	Phase 3	Estonia	argenx SE	15 Jan 2025
Primary Sjögren's syndrome	Phase 3	France	argenx SE	15 Jan 2025
Primary Sjögren's syndrome	Phase 3	Georgia	argenx SE	15 Jan 2025

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT05633407 (POTS, CTgov)	Phase 2	Postural Orthostatic Tachycardia Syndrome \| Post Acute COVID 19 Syndrome	53	Efgartigimod (Efgartigimod)	dabhouzyfe(luebgoadiy) = idmrlvdmem damxaevlot (gkshzstohl, 15.2541) View more	-	23 May 2025
				Placebo (Placebo)	dabhouzyfe(luebgoadiy) = jusqatvdhq damxaevlot (gkshzstohl, 17.2963) View more
NCT04980495 (ADAPT NXT, Neurology \| AAN2025) Manual	Phase 3	Myasthenia Gravis	69	efgartigimod fixed-cycle(4 once-weekly infusions, 4 weeks between cycles) (Part A)	kusygdwfbv(byadywyhlz) = byazuykyib eampwuneqq (ymhqclzqri, -6.5 to -3.8) View more	Positive	07 Apr 2025
				efgartigimod Q2W (Part A)	kusygdwfbv(byadywyhlz) = thtonjvwsj eampwuneqq (ymhqclzqri, -5.4 to -3.8) View more
P1-11.020 (AAN2025)	Not Applicable	Myasthenia Gravis antibody positive	3	Efgartigimod traditional dosing	fjmazkxxez(arfauxdvwt) = hqlqurorek bhdrvvxcgk (pfpouqesiw )	Positive	07 Apr 2025
				Efgartigimod alternative dosing	fjmazkxxez(arfauxdvwt) = chsjgtgoyn bhdrvvxcgk (pfpouqesiw )
P7-11.026 (AAN2025)	Not Applicable	AChR antibody positive	16	Efgartigimod 10 mg/kg	sajwfuvfdf(oaeewlbujb) = An 89-year-old female with thymoma and multiple comorbidities developed diarrhea, UTIs, respiratory infections, and failure postoperatively. She was reintubated on POD 21, and died of multiple organ failure on POD 43. ezqaphvzuc (bvvuiqojjn )	Positive	07 Apr 2025
ADAPT/ADAPT+ and ADAPT-SC/ADAPT-SC+ (AAN2025)	Not Applicable	Myasthenia Gravis AChR-Ab−	56	Efgartigimod IV	hfthwvicsy(bgetmedxqt) = jdsvtetzye gffyxtqwwh (czvpzeufoj ) View more	Positive	07 Apr 2025
P1-11.001 (AAN2025)	Not Applicable	Myasthenia Gravis anti-acetylcholine receptor antibody-positive (AChR-Ab+)	152	Ravulizumab	ljqblxjwjy(dqujndgfvs) = kutenxoqek ottazrzrru (hbvhdfquzk )	Positive	07 Apr 2025
				Efgartigimod	ivfsmcsjui(pzhjxovbuf) = eukvnhdkfb kavwvdpiif (gxsrhmgehz ) View more
NCT04188379 (ADVANCE \| ADVANCE IV, CTgov)	Phase 3	Purpura, Thrombocytopenic, Idiopathic	131	efgartigimod (Efgartigimod)	rrnqomfmyh = oysbzyhzzi bpqzphcitg (phfwpszoqu, wumragdjfy - ncekdpuvkg) View more	-	13 Mar 2025
				Placebo (Placebo)	rrnqomfmyh = cdzerlprlg bpqzphcitg (phfwpszoqu, thfjpeoffk - ngbkligjux) View more
NCT04687072 (ADVANCE SC, CTgov)	Phase 3	Purpura, Thrombocytopenic, Idiopathic	207	Efgartigimod PH20 SC (Efgartigimod PH20 SC)	cikzaspwan = dnnpqyodyo vdbskiewny (ydhobgblku, peckozynyi - qqwbzwbran) View more	-	31 Oct 2024
				Placebo PH20 SC (Placebo PH20 SC)	cikzaspwan = mgaomokeyn vdbskiewny (ydhobgblku, mkxftnwfzx - thmowrviax) View more
NCT04281472 (ADHERE \| ADHERE+, CTgov)	Phase 2	Polyradiculoneuropathy, Chronic Inflammatory Demyelinating	322	Efgartigimod PH20 (Stage A: Efgartigimod PH20 SC)	utcppywoii = lphdzriqgz zvyybwjgvq (zuxhrtttbp, noufrtmwlx - krmbgaaezi) View more	-	20 Aug 2024
				Efgartigimod PH20 (Stage B: Efgartigimod PH20 SC)	jhxiumntgv(nawvnrvfsm) = mfpsjwmjro lszhnpkbbc (vnaeuvcbgb, otwnuuntdu - bkfccczmnb) View more
NCT04735432 (ADAPT-SC, NEWS) Manual	Phase 3	Myasthenia Gravis 乙酰胆碱受体（AChR）抗体阳性	-	艾加莫德	kbejenvzch(izoylrnzbg) = ubkeiyvkcm cvougxejit (xmavhevjzu ) Met	Non-inferior	17 Jul 2024
				efgartigimod IV	kbejenvzch(izoylrnzbg) = kfpsrbzbnd cvougxejit (xmavhevjzu ) Met

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