Key Takeaways:
FDA approved Johnson & Johnson's IMAAVY as the first wAIHA treatment after roughly three times as many patients hit durable hemoglobin response versus placebo.
"For the first time, our community has a treatment specifically for our disease," Karen Jones, president and executive director of patient group wAIHA Warriors, said.
The clearance covers adults and pediatric patients 12 and older currently or previously treated with corticosteroids. In the Phase 2/3 ENERGY study, patients on the approved 30 mg/kg dose given every four weeks showed a mean hemoglobin increase of 1 g/dL at week one, with a median time to first response of 4.1 weeks versus 12.1 weeks for placebo. IMAAVY also delivered a 3.5-point higher mean FACIT-Fatigue score at week 24, and the most common adverse reactions were peripheral edema, diarrhea and fever.
"More patients treated with IMAAVY achieved a durable hemoglobin response compared with placebo — meaning their red blood cell levels went up and stayed up," David Kuter, a physician at Massachusetts General Hospital and professor of medicine at Harvard Medical School, said.
The approval marks IMAAVY's second US clearance after generalized myasthenia gravis in April 2025, extending Johnson & Johnson's push into FcRn-blocker territory where it competes with Argenx's Vyvgart Hytrulo and UCB's Rystiggo. Warm autoimmune hemolytic anemia affects roughly one to three new people per 100,000 each year, with about one in 8,000 living with the condition.
Johnson & Johnson acquired nipocalimab through its $6.5 billion purchase of Momenta Pharmaceuticals in 2020. The ENERGY trial randomized 115 adults, and the drug's safety profile in wAIHA was consistent with that seen in generalized myasthenia gravis. Full results were presented at the European Hematology Association meeting in June, and patients could enter an open-label extension for up to 144 weeks. The company is offering the IMAAVY withMe support program covering education, nurse navigation and cost help regardless of insurance type.
"Today's announcement marks the second approval for IMAAVY and is an extraordinary milestone for people living with warm autoimmune hemolytic anemia," David M. Lee, global immunology therapeutic area head at Johnson & Johnson, said.
The approval gives Johnson & Johnson a first-mover position in an underserved rare-disease market, adding to a drug the company has suggested could reach $5 billion in annual sales. Investors will watch launch uptake and further label expansions as nipocalimab advances in Sjogren's disease, lupus and hemolytic disease of the fetus and newborn.
This article is for informational purposes only and does not constitute investment advice.
