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Data from the pivotal ENERGY trial showed IMAAVY® produced a rapid and durable hemoglobin responsea in wAIHA
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Currently no FDA-approved therapies are available for wAIHA, a rare, heterogeneous, life-threatening disease in which pathogenic immunoglobulin (IgG) autoantibodies attach to and destroy red blood cells, leading to debilitating anemia
SPRING HOUSE, Pa., (February 24, 2026) – Johnson & Johnson (NYSE: JNJ) today announced the submission of a supplemental Biologics License Application (sBLA) to the U.S. Food and Drug Administration (FDA), seeking approval of IMAAVY® (nipocalimab-aahu) as the first-ever treatment for patients with warm autoimmune hemolytic anemia (wAIHA).b This rare and serious autoantibody disease affects approximately 1 in 8,000 in the United States and currently has no approved treatments despite substantial unmet need.1 The condition is associated with significant morbidity and mortality, with those living with the disease found to experience a 20-30% higher risk of death.2
“People living with warm autoimmune hemolytic anemia face a serious, life-threatening disease with no approved treatment options and a high risk of complications, including profound chronic fatigue, transfusion dependence, and organ failure,” said David M Lee, M.D., Ph.D., Global Immunology Therapeutic Area Head, Johnson & Johnson. “The submission of this sBLA represents an important milestone for the wAIHA community and underscores our commitment to advancing targeted, immunoselective therapies that can deliver meaningful, rapid improvement for these patients.”
wAIHA occurs when harmful immunoglobulin G (IgG) autoantibodies attach to and destroy red blood cells – leading to anemia.3 IMAAVY® is designed to selectively block the neonatal Fc receptor (FcRn), a key regulator of IgG recycling.4 By reducing circulating IgG, including autoantibodies, IMAAVY® targets the underlying cause of disease while preserving critical immune functions, including some humoral B-cell responses to new infections.
The sBLA submission is supported by the Phase 2/3 ENERGY multicenter, randomized, double-blind, placebo-controlled study (NCT04119050) evaluating IMAAVY® in adults living with wAIHA. The data showed that more patients treated with nipocalimab achieved the stringent primary endpoint of a durable hemoglobin response compared with placebo. A durable response was defined as achieving a hemoglobin level above 10 g/dL and an increase of at least 2 g/dL for at least 28 days, without the need for rescue therapy.5
In addition to a rapid and durable improvement in hemoglobin, more patients treated with IMAAVY® experienced rapid and sustained improvement in fatigue as assessed by FACIT-Fatigue, an outcome of significant importance to people living with wAIHA.5
“The ENERGY study demonstrated clinically meaningful results in adults living with warm autoimmune hemolytic anemia,” said Bruno Fattizzo, M.D., Assistant Professor at the Department of Oncology and Hematology-Oncology, Università degli Studi di Milanoc. “These results provide a strong rationale for the potential of IMAAVY to rapidly improve fatigue and provide durable hemoglobin response while maintaining favorable tolerability.”
IMAAVY® was generally well tolerated in ENERGY, with no new safety signals identified and a safety profile consistent with the IMAAVY® label.5,6 IMAAVY® was approved in the United States in April 2025 for the treatment of generalized myasthenia gravis (gMG) in adult and pediatric patients 12 years of age and older who are acetylcholine receptor (AChR) or muscle-specific kinase (MuSK) antibody positive.6
The full results of the ENERGY trial are forthcoming.
Editor’s notes:
Durable hemoglobin response = hemoglobin concentration ≥10 g/dL and an increase from baseline in Hgb ≥2 g/dL for at least 28 days
IMAAVY® is not approved in wAIHA
Dr. Fattizzo has served as a consultant to J&J; he has not been paid for any media work
ABOUT THE ENERGY TRIAL
ENERGY (NCT04119050) is a multicenter, randomized, double-blind, placebo-controlled Phase 2/3 study evaluating the efficacy and safety of nipocalimab compared with placebo, followed by an open-label extension period, in adults living with wAIHA.5
ABOUT WARM AUTOIMMUNE HEMOLYTIC ANEMIA (wAIHA)
Warm autoimmune hemolytic anemia (wAIHA) is a rare, life-threatening condition where autoantibodies attach to and destroy red blood cells (RBCs), resulting in anemia. Approximately 1-3 new people per 100,000 are affected by wAIHA per year, and about 1 in 8,000 individuals are living with the condition.1,7 This condition affects both women and men, and can affect people at any age with incidence increasing over the age of 50.8,9 Additionally, people with wAIHA are at increased risk of other serious complications such as venous thrombotic events, acute renal failure, and infection.10
There are no Food and Drug Administration (FDA)-approved drugs indicated for wAIHA, and treatment typically consists of unapproved corticosteroids, broad immunosuppressants, and B-cell directed therapies.7 With an unmet need for treatment in wAIHA, novel therapies like nipocalimab that can deliver meaningful improvement to patients is critical.9
ABOUT IMAAVY® (nipocalimab-aahu)
IMAAVY® 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. IMAAVY® is currently approved for the treatment of gMG in adults and pediatric patients 12 years of age and older who are AChR or MuSK antibody positive.6
Nipocalimab is being investigated across three key segments in the autoantibody space including Rheumatologic diseases, Rare Autoantibody diseases, and Maternal Fetal 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.11,12,13,14,15,16,17,18,19,20
The U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) have granted several key designations to nipocalimab including:
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U.S. FDA Fast Track designation in hemolytic disease of the fetus and newborn (HDFN) and warm autoimmune hemolytic anemia (wAIHA) in July 2019, gMG in December 2021, fetal and neonatal alloimmune thrombocytopenia) FNAIT in March 2024 and Sjögren’s disease (SjD) in March 2025
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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
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U.S. FDA Breakthrough Therapy designation for HDFN in February 2024 and for SjD in November 2024
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U.S. FDA granted Priority Review in gMG in Q4 2024
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EU EMA Orphan medicinal product designation for HDFN in October 2019 and FNAIT in April 2025
The legal manufacturer for IMAAVY® is Janssen Biotech, Inc.
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.
Footnotes:
1 Tranekær S, Hansen DL, Frederiksen H. Epidemiology of secondary warm autoimmune haemolytic anaemia-A systematic review and meta-analysis. J Clin Med. 2021 Mar 17;10(6):1244. doi:10.3390/jcm10061244. PMID: 33802848; PMCID: PMC8002719.
2 Jackson L, Zhdanava M, Pesa J, Boonmak P, Chen G, Liu D, et al. Mortality associated with warm autoimmune hemolytic anemia among Medicare beneficiaries. Blood. 2025;2694, 146 (Suppl 1):2694. https://doi.org/10.1182/blood-2025-2694
3 National Organization for Rare Disorders, Warm autoimmune Hemolytic Anemia. Available at: https://rarediseases.org/rare-diseases/warm-autoimmune-hemolytic-anemia/. Last accessed: February 2026.
4 Cossu M et al. A randomized, open-label study on the effect of nipocalimab vaccine response in healthy participants. Presentation at American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) Annual Meeting. October 2024.
5 ClinicalTrials.gov Identifier: NCT04119050. Available at: https://www.clinicaltrials.gov/study/NCT04119050
6 IMAAVY® U.S. Prescribing Information.
7 Sudulagunta SR, et al. Warm Autoimmune Hemolytic Anemia: Clinical Profile and Management. J Hematol. 2017 Mar; 6(1): 12–20. Published online 2017 Mar 21. doi: 10.14740/jh303w.
8 National Organization for Rare Disorders, Warm autoimmune Hemolytic Anemia. Available at: https://rarediseases.org/rare-diseases/warm-autoimmune-hemolytic-anemia/. Last accessed: February 2026.
9 Cherif H, Cai ., Crivera, C, Leon A, Rahman I, Leval A, Noel W, Kjellander C. Overall survival and treatment patterns among patients with warm wutoimmune hemolytic anemia in Sweden: A nationwide population-based. 2024.
10 Fattizzo B, Barcellini W. New therapies for the treatment of warm autoimmune hemolytic anemia. Transfusion Medical Reviews. 2022;36(4). https://doi.org/10.1016/j.tmrv.2022.08.001
11 ClinicalTrials.gov Identifier: NCT04951622. Available at: https://clinicaltrials.gov/ct2/show/NCT04951622. Last accessed: February 2026.
12 ClinicalTrials.gov. NCT03842189. Available at: https://clinicaltrials.gov/ct2/show/NCT03842189. Last accessed: February 2026.
13 ClinicalTrials.gov Identifier: NCT05327114. Available at: https://www.clinicaltrials.gov/study/NCT05327114. Last accessed: February 2026.
14 ClinicalTrials.gov Identifier: NCT04119050. Available at: https://clinicaltrials.gov/study/NCT04119050. Last accessed: February 2026.
15 ClinicalTrials.gov Identifier: NCT05379634. Available at: https://clinicaltrials.gov/study/NCT05379634. Last accessed: February 2026.
16 ClinicalTrials.gov Identifier: NCT05912517. Available at: https://www.clinicaltrials.gov/study/NCT05912517. Last accessed: February 2026.
17 ClinicalTrials.gov Identifier: NCT04968912. Available at: https://clinicaltrials.gov/study/NCT04968912. Last accessed: February 2026.
18 ClinicalTrials.gov Identifier: NCT04882878. Available at: https://clinicaltrials.gov/study/NCT04882878. Last accessed: February 2026.
19 ClinicalTrials.gov Identifier: NCT06449651. Available at: https://clinicaltrials.gov/study/NCT06449651. Last accessed: February 2026.
20 ClinicalTrials.gov Identifier: NCT06533098 Available at: https://clinicaltrials.gov/study/NCT06533098. Last accessed: February 2026.