Nipocalimab-aahu

Johnson & Johnson (NYSE: JNJ) announced receipt of US FDA Priority Review for the supplemental Biologics License Application (sBLA) for Imaavy (nipocalimab-aahu), an anti-neonatal Fc receptor (FcRn) monoclonal antibody, for the treatment of warm autoimmune hemolytic anemia (wAIHA). The filing marks the first time any therapy has received Priority Review from the agency for this condition. Priority Review shortens the FDA’s standard ten-to-twelve-month review timeline to approximately six months for medicines that may offer meaningful improvements in safety or effectiveness for serious conditions. Nipocalimab had previously received FDA Fast Track designation for wAIHA in July 2019 and Orphan Drug designation for the same indication in December 2019. The sBLA is supported by data from the ENERGY trial, a multicenter, randomized, double-blind, placebo-controlled Phase 2/3 study in adults with wAIHA. The primary endpoint was durable hemoglobin response, defined as hemoglobin concentration of at least 10 g/dL and an increase from baseline of at least 2 g/dL sustained for a minimum of 28 days without rescue therapy. Johnson & Johnson reported that more patients treated with nipocalimab met this endpoint than those receiving placebo, and that fatigue — measured by the Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) scale — also improved. The company has stated that full ENERGY results will be presented at a forthcoming medical conference; precise response rates, effect sizes, and p-values have not yet been disclosed in any public source identified at the time of this report. Nipocalimab blocks FcRn, a receptor that recycles immunoglobulin G (IgG) antibodies and extends their half-life in circulation. By occupying FcRn, the drug accelerates IgG catabolism, reducing total circulating IgG including the pathogenic autoantibodies that drive red blood cell destruction in wAIHA. The mechanism is selective in that it does not directly deplete B cells or broadly suppress innate immune function, which distinguishes it from the corticosteroids, rituximab, and broad immunosuppressants that currently constitute off-label standard of care in this disease. Imaavy is already FDA-approved — the agency cleared the product on April 29, 2025 for generalized myasthenia gravis (gMG) in adults and pediatric patients aged 12 years and older who are anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive. The wAIHA sBLA therefore represents a label expansion for an approved molecule rather than an initial market entry. Research context wAIHA is a rare condition in which IgG autoantibodies bind to red blood cell surface antigens, triggering their destruction by the reticuloendothelial system and producing hemolytic anemia of variable severity. Incidence is estimated at one to three new cases per 100,000 persons annually, with a prevalence of approximately one in 8,000. The condition affects both sexes across all age groups, though incidence rises after age 50. Complications extend beyond anemia and include venous thrombotic events, acute renal failure, and infection risk. There are currently no FDA-approved drugs indicated for wAIHA. Nipocalimab is also in development across a broader portfolio of IgG-mediated diseases. Active programs include studies in hemolytic disease of the fetus and newborn (HDFN), for which the molecule holds FDA Breakthrough Therapy designation granted in February 2024; fetal and neonatal alloimmune thrombocytopenia (FNAIT); Sjögren’s disease, which received Breakthrough Therapy designation in November 2024; systemic lupus erythematosus; and chronic inflammatory demyelinating polyneuropathy (CIDP), among others. The FDA granted Priority Review for the gMG application in Q4 2024, leading to the April 2025 approval. The PDUFA action date for the wAIHA sBLA has not been disclosed. Given the FDA’s approximately six-month Priority Review timeline, a decision could fall in late 2026, though the agency has not publicly confirmed the target date. Your email address will not be published. Required fields are marked * Comment * Name * Email * Website

Through 120 weeks of follow-up, nipocalimab delivered sustained clinical improvements and reductions in total IgG in antibody-positive adult patients including anti-AChR+ and anti-MuSK+ Patients achieving sustained minimal symptom expression (MSE) experienced greater improvements in quality of life than those with transient MSE in a post-hoc analysis of the Phase 3 study EPIC, a head-to-head study, will compare nipocalimab versus another FcRn blocker in generalised myasthenia gravis a BEERSE, BELGIUM, April 23, 2026 (GLOBE NEWSWIRE) -- Johnson & Johnson today announced new data from the Phase 3 Vivacity-MG3 study and ongoing open label extension (OLE) in a broad population of antibody-positive (including anti-AChR+ b and anti-MuSK+ c ) adults with generalised myasthenia gravis (gMG) reinforcing the efficacy, sustained disease control, and safety pro IMAAVY ® ▼(nipocalimab). 1 These data are among the seven abstracts Johnson & Johnson is presenting at the American Academy of Neurology (AAN) 2026 Meeting in Chicago, Illinois. “For people living with gMG, consistent and durable symptom control is the central goal of treatment,” said Constantine Farmakidis M.D., Associate Professor of Neurology at the University of Kansas Medical Center d . “These long-term results, now extending to beyond two years, provide further evidence that disease control, as initially observed in the nipocalimab phase 3 pivotal study, can be sustained, and add to the body of evidence that may help guide clinical decision-making.” Sustained disease control is a key treatment objective in gMG, as long-term maintenance of low disease activity can help prevent exacerbations, reduce treatment burden and support meaningful function outcomes. 1 , 2 , 3 , 4 , 5 , 6 In addition, new post-hoc analyses explore the clinical relevance of sustained minimal symptom expression (MSE), an emerging patient-centric treatment goal that reflects minimal day-to-day disease impact for people living with gMG. 2 , 3 Long-Term Data from OLE Phase After the 24-week double-blind phase of the study, patients entered the ongoing OLE phase, with the latest results reflecting a maximum of 120 weeks of observation – among the longest follow-up periods reported for any FcRn blocker study in gMG. 1 At 96 weeks in the OLE, nipocalimab demonstrated: Sustained improvements in MG-ADL e and QMG f scores over time, with mean reductions of 6.47 g points on the total MG-ADL (standard error [SE]=1.20, p<0.001) and 5.97 g points (SE=1.28, p<0.001) on the total QMG scales – measures of MG symptom impact on daily living and muscle strength. 1 , 7 , 8 Half of patients achieved MSE (n=153) and nearly one-third (32%) achieved sustained MSE for at least 8 weeks on nipocalimab treatment. 1 Incremental reduction of corticosteroid use was also observed through the OLE, with 57% of patients reaching low doses of ≤10 or ≤5 mg/day. 1 Greater than 64% g reduction in total immunoglobulin G (IgG), including pathogenic IgG autoantibodies, the underlying driver of disease (n=31, standard deviation [SD]=12.91). 1 There were no unexpected adverse events (AEs) during the OLE. The most common AEs associated with nipocalimab include muscle spasms, peripheral oedema, increased lipids and decreased serum albumin. 1 , 9 , h “These long-term findings for nipocalimab reflect continued momentum in transforming the treatment landscape for people living with generalised myasthenia gravis,” said Mark Graham, Therapeutic Area Head, Immunology, Europe, Middle East and Africa Johnson & Johnson. “Evidence demonstrating sustained disease control alongside sustained improvements in quality of life underscores our progress toward more effective treatment options. We remain committed to advancing innovative approaches that address persistent unmet needs and improve long-term outcomes for patients.” Minimal Symptom Expression Data from Double-Blind Phase A new post-hoc analysis from the 24-week double-blind portion of the study evaluated the impact of sustained MSE on quality of life (based on MG‑QoL‑15r i measure): 2 Adults who received nipocalimab plus standard of care (SOC) j were four times more likely to reach sustained MSE, defined as achieving an MG-ADL score of 0 or 1 and maintaining it for at least 8 weeks ([95% CI]: 4.35 [1.37, 13.81], p=0.013), compared to those randomised to placebo. 2 Patients who reached this level at Week 24 and sustainment of symptom control experienced greater improvements in day‑to‑day quality of life ([95% CI]: −12.7, n=19, p<0.001), compared with those with improvements that were not similarly sustained (−8.7, n=14, p=0.016), or among those who did not attain MSE (−4.1, n=96, p<0.001). 2 Editor’s Notes: Study details, including participating locations and eligibility criteria, are available on ClinicalTrials.gov. 10 AChR+= anti-acetylcholine receptor positive antibody MuSK+= anti-muscle specific tyrosine kinase positive antibody Dr. Constantine Farmakidis M.D. has provided consulting, advisory, and speaking services to Johnson & Johnson. He has not been paid for any media work. MG-ADL (Myasthenia Gravis – Activities of Daily Living) provides a rapid clinical assessment of the patient's recall of symptoms impacting activities of daily living, with a total score range of 0 to 24; a higher score indicates greater symptom severity. 7 QMG (Quantitative Myasthenia Gravis) is a 13-item assessment by a clinician that quantifies MG disease severity. The total QMG score ranges from 0 to 39, where higher scores indicate greater disease severity. 8 Results reflect patients receiving nipocalimab and SOC throughout both the 24-week double-blind phase and OLE phase of the study. 1 The overall incidence of AEs, serious adverse events (SAEs) and AEs leading to discontinuation were similar to that in the placebo plus SOC group. 1 Specifically, 46% of patients (n=64) treated with nipocalimab plus SOC experienced AEs, matched by 45% (n=62) in the placebo plus SOC group. 1 SAEs were reported by 3% of patients (n=5) in the nipocalimab plus SOC group compared to 8% (n=11) in the placebo plus SOC group. 1 As measured by MG-QoL-15r (Myasthenia Gravis Quality of Life 15-item Scale – Revised), a scale designed to assess important aspects of the patient's experience related to MG. 2 Standard of care was defined as a stable dose of current gMG treatment, including acetylcholinesterase inhibitors, glucocorticosteroids or immunosuppressants (e.g, azathioprine, mycophenolate mofetil or mycophenolic acid, methotrexate, ciclosporin, tacrolimus, or cyclophosphamide). 11 ABOUT GENERALISED MYASTHENIA GRAVIS (gMG) Myasthenia gravis (MG) is an autoantibody disease in which the immune system mistakenly makes antibodies (e.g., anti-acetylcholine receptor [AChR], anti-muscle-specific tyrosine kinase [MuSK]), which target proteins at the neuromuscular junction and can block or disrupt normal signaling from nerves to muscles, thus impairing or preventing muscle contraction. 12 , 13 The disease impacts between 56,000 and 123,000 people in Europe and an estimated 700,000 people worldwide. 14 , 15 The disease affects both men and women and occurs across all ages, racial and ethnic groups, but most frequently starts in young women and older men. 14 Roughly 50 percent of individuals diagnosed with MG are women, and about one in five of those women are of child-bearing potential. 16 , 17 , 18 Approximately 10 to 15% of new cases of MG are diagnosed in paediatric patients 12-17 years of age. 17 , 19 , 20 , 21 Among juvenile MG patients, girls are affected more often than boys with over 65% of paediatric MG cases in the EU diagnosed in girls. 22 , 23 , 24 Initial disease manifestations are usually eye-related but approximately 85% of MG patients experience additional advancements to the disease manifestations – referred to as generalised myasthenia gravis (gMG). 25 , 26 , 27 , 28 , 29 , 30 This is characterised by severe muscle weakness and difficulties in speech and swallowing. 26 , 28 , 29 , 30 , 31 , 32 Vulnerable gMG populations, such as paediatric patients, have more limited therapeutic options. 32 ABOUT THE PHASE 3 VIVACITY-MG3 STUDY The Phase 3 Vivacity-MG3 study ( NCT04951622 /2023-504152-97) was specifically designed to measure sustained efficacy and safety with consistent dosing in this unpredictable chronic condition where unmet need remains high. 14 , 33 , 34 Antibody positive or negative adult gMG patients with insufficient response (MG-ADL ≥6) to ongoing SOC therapy were identified and 199 patients, 153 of whom were antibody positive, enrolled in the 24-week double-blind placebo-controlled trial. 33 , 34 Randomisation was 1:1, nipocalimab plus current SOC (30 mg/kg IV loading dose followed by 15 mg/kg every two weeks) or placebo plus current SOC. 34 Baseline demographics were balanced across arms (77 nipocalimab, 76 placebo) . 34 The primary efficacy endpoint was the comparison of the mean change from baseline to Weeks 22, 23, and 24 between treatment groups in the MG-ADL total score. 33 A key secondary endpoint included change in Quantitative Myasthenia Gravis (QMG) score. 33 Long-term safety and efficacy were further assessed in an ongoing open-label extension (OLE) phase. 1 ABOUT IMAAVY ® (nipocalimab) Nipocalimab is an immunoselective treatment designed to target, bind with high affinity, and block FcRn, reducing circulating IgG antibodies that drive disease while also preserving key immune functions. 35 , 36 , 37 Nipocalimab is currently approved in the European Union as an add-on to standard therapy for the treatment of gMG, in adults and adolescent patients aged 12 years of age and older who are AChR or MuSK antibody positive. 9 , 38 Nipocalimab is being investigated across three key segments in the autoantibody space including Rheumatologic diseases, Rare Autoantibody diseases, and Maternal Foetal diseases mediated by maternal alloantibodies, in which blockade of IgG binding to FcRn in the placenta is believed to limit transplacental transfer of maternal alloantibodies to the fetus. 33 , 39 , 40 , 41 , 42 , 43 , 44 , 45 , 46 , 47 The U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) have granted several key designations to nipocalimab including: EU EMA Orphan medicinal product designation for haemolytic disease of the foetus and newborn (HDFN) in October 2019 and foetal and neonatal alloimmune thrombocytopenia (FNAIT) in April 2025 U.S. FDA Fast Track designation in HDFN and warm autoimmune haemolytic anaemia (wAIHA) in July 2019, gMG in December 2021, FNAIT in March 2024, Sjögren’s disease (SjD) in March 2025, and systemic lupus erythematosus (SLE) in January 2026 U.S. FDA Orphan drug status for wAIHA in December 2019, HDFN in June 2020, gMG in February 2021, chronic inflammatory demyelinating polyneuropathy (CIDP) in October 2021 and FNAIT in December 2023 U.S. FDA Breakthrough Therapy designation for HDFN in February 2024 and for SjD in November 2024 U.S. FDA granted Priority Review in gMG in Q4 2024 For a full list of adverse events and information on dosage and administration, contraindications and other precautions when using nipocalimab please refer to the Summary of Product Characteristics . ▼ In line with EU regulations for new medicines, nipocalimab is subject to additional monitoring. ABOUT JOHNSON & JOHNSON At Johnson & Johnson, we believe health is everything. Our strength in healthcare innovation empowers us to build a world where complex diseases are prevented, treated, and cured, where treatments are smarter and less invasive, and solutions are personal. Through our expertise in Innovative Medicine and MedTech, we are uniquely positioned to innovate across the full spectrum of healthcare solutions today to deliver the breakthroughs of tomorrow and profoundly impact health for humanity. Learn more at Follow us at CAUTIONS CONCERNING FORWARD-LOOKING STATEMENTS This press release contains “forward-looking statements” as defined in the Private Securities Litigation Reform Act of 1995 regarding product development and the potential benefits and treatment impact of IMAAVY. The reader is cautioned not to rely on these forward-looking statements. These statements are based on current expectations of future events. If underlying assumptions prove inaccurate or known or unknown risks or uncertainties materialize, actual results could vary materially from the expectations and projections of Johnson & Johnson. Risks and uncertainties include, but are not limited to: challenges and uncertainties inherent in product research and development, including the uncertainty of clinical success and of obtaining regulatory approvals; uncertainty of commercial success; manufacturing difficulties and delays; competition, including technological advances, new products and patents attained by competitors; challenges to patents; product efficacy or safety concerns resulting in product recalls or regulatory action; changes in behavior and spending patterns of purchasers of health care products and services; changes to applicable laws and regulations, including global health care reforms; and trends toward health care cost containment. A further list and descriptions of these risks, uncertainties and other factors can be found in Johnson & Johnson’s most recent Annual Report on Form 10-K, including in the sections captioned “Cautionary Note Regarding Forward-Looking Statements” and “Item 1A. Risk Factors,” and in Johnson & Johnson’s subsequent Quarterly Reports on Form 10-Q and other filings with the Securities and Exchange Commission. Copies of these filings are available online at or on request from Johnson & Johnson. Johnson & Johnson does not undertake to update any forward-looking statement as a result of new information or future events or developments. Source: Johnson & Johnson For European medical and trade media only. Not for distribution in the UK, Ireland and BeNeLux. CP-576128 April 2026 ### REFERENCES 1 Antozzi, C et al., Long-term Safety and Efficacy of Nipocalimab: Approximately 2 Years Follow-Up Results from the Open-Label Extension Phase of Vivacity-MG3 Study. Abstract #9.018 for poster presentation at 2026 American Academy of Neurology Congress. April 2026. 2 Vicente, E et al., Quality of Life in Patients with Generalized Myasthenia Gravis​ Achieving Sustained vs Transient Minimal Symptom Expression in the Phase 3 Vivacity-MG3 Trial. Abstract #9.007 for poster presentation at 2026 American Academy of Neurology Congress. April 2026 3 Uzawa, A et al. Minimal symptom expression achievement over time in generalized myasthenia gravis. Acta Neurol Belg. 2023;123(3):979-982. 4 Nelke, C et al. Independent risk factors for myasthenic crisis and disease exacerbation in a retrospective cohort of myasthenia gravis patients. J Neuroinflammation. 2022;12;19(1):89. 5 Banner, H et al. Myasthenia gravis in pregnancy: Systematic review and case series. Obstet Med. 2022;15(2):108- 117. 6 Barraud, C et al. Clinical features and evolution of juvenile myasthenia gravis in a French cohort. Muscle Nerve. 2018;57(4):603-609. 7 Wolfe GI. 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Johnson & Johnson receives European Commission approval of IMAAVY ® (nipocalimab), a new FcRn blocker offering sustained disease control in a broad population of people living with generalised myasthenia gravis (gMG). Available at: . Last accessed: April 2026. 39 ClinicalTrials.gov. NCT03842189. Available at: . Last accessed: April 2026. 40 ClinicalTrials.gov Identifier: NCT05327114. Available at: . Last accessed: April 2026. 41 ClinicalTrials.gov Identifier: NCT04119050. Available at: . Last accessed: April 2026. 42 ClinicalTrials.gov Identifier: NCT05379634. Available at: . Last accessed: April 2026. 43 ClinicalTrials.gov Identifier: NCT05912517. Available at: . Last accessed: April 2026. 44 ClinicalTrials.gov Identifier: NCT04968912. Available at: . Last accessed: April 2026. 45 ClinicalTrials.gov Identifier: NCT04882878. Available at: . Last accessed: April 2026. 46 ClinicalTrials.gov Identifier: NCT06449651. Available at: . Last accessed: April 2026. 47 ClinicalTrials.gov Identifier: NCT06533098. Available at: . Last accessed: April 2026. CONTACT: Media contact: Alexandra Nisipeanu adridean@its.jnj.com Investor contact: Jessica Margevich investor-relations@its.jnj.com