Johnson & Johnson 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. 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.

"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.³IMAAVY^® 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.

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 Milano.^c "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.

The full results of the ENERGY trial are forthcoming.