Approval of Imaavy (Nipocalimab) and its Impact on Patients With Warm Autoimmune Hemolytic Anemia

Karen A. Jones, EdD, President and CEO of wAIHA Warriors, discusses the approval of Imaavy (nipocalimab) and its impact on patients with warm autoimmune hemolytic anemia (wAIHA). wAIHA is a rare hematologic autoimmune condition characterized by antibodies that attack red blood cells and lead to hemolytic anemia. Symptoms may include unusual weakness and fatigue that make daily life difficult, tachycardia, breathing difficulties, jaundice, dark urine and/or splenomegaly. The cause of wAIHA is unknown. The US Food and Drug Administration (FDA) recently approved nipocalimab for the treatment of wAIHA in patients 12 years of age and older currently or previously treated with corticosteroids. Dr. Jones describes the approval as a major milestone that provides patients with treatment options and hope for the future of wAIHA. Nipocalimab is an immunoselective neonatal fragment crystallizable receptor (FcRn) blocker designed to target and reduce pathogenic immunoglobulin G (IgG)  autoantibodies while preserving B-cell function. It  is the first  FDA approved treatment for wAIHA. The approval was based on the pivotal phase 2/3 ENERGY study (NCT04119050), where patients treated with nipocalimab  demonstrated durable hemoglobin response. A mean increase in hemoglobin of 1 g/DL at week 1 and improvement in FACIT-Fatigue score at week 24 were also observed. Nipocalimab’s safety profile cwas onsistent with the established safety profile in generalized myasthenia gravis (gMG). The most common adverse reactions in patients with wAIHA treated with nipocalimab were peripheral edema, diarrhea, and fever. Dr. Jones hopes that this treatment approval will lead to greater recognition and understanding of wAIHA, especially among physicians. This in turn can help patients get earlier referrals to hematologists and diagnoses. Additionally, Dr. Jones and patients remain hopeful for further research and a greater awareness of symptom burden. For more information on the approval of nipocalimab, visit https://www.jnj.com/media-center/press-releases/fda-approves-imaavy-nipocalimab-aahu-as-first-ever-treatment-for-warm-autoimmune-hemolytic-anemia-waiha-representing-a-landmark-advancement-for-patients To learn more about wAIHA and other rare hematologic conditions, visit https://checkrare.com/diseases/hematologic-disorders/ **Rare Discussions is produced by CheckRare**, the leading multimedia platform dedicated to advancing education, awareness, and innovation across the rare disease community. Explore additional physician interviews, podcasts, CME activities, and rare disease resources at **CheckRare.com**. Subscribe to the **CheckRare Podcast Network** for expert conversations, weekly news, accredited education, and the latest advances across the rare disease community. Part of the **CheckRare Podcast Network**: Trusted conversations, news, education, and expert insights across the rare disease community.