Johnson & Johnson Secures FDA Priority Review for IMAAVY® in wAIHA

Johnson & Johnson has announced that the U.S. Food and Drug Administration (FDA) has granted Priority Review to the supplemental Biologics License Application (sBLA) for nipocalimab-aahu (IMAAVY®) for the treatment of warm autoimmune hemolytic anemia (wAIHA). This reflects the current absence of FDA-approved therapies specifically indicated for warm autoimmune hemolytic anemia (wAIHA)

The FDA’s decision is supported by data from the Phase 2/3 ENERGY clinical trial (NCT04119050). In this study, patients treated with nipocalimab, administered as an intravenous infusion, demonstrated a durable hemoglobin response compared with those receiving placebo.

A durable response was defined as achieving a hemoglobin concentration ≥10 g/dL and an increase from baseline in hemoglobin ≥2 g/dL for at least 28 days. Improvements in fatigue were observed, an important outcome given the burden of symptoms associated with wAIHA.

The FDA’s Priority Review designation is reserved for medicines that have the potential to provide significant improvements in safety or effectiveness in treating serious diseases. This designation reduces the standard review timeline to approximately six months, enabling faster evaluation of promising therapies.

Notably, IMAAVY is among the first therapies in wAIHA to receive this designation, highlighting both the severity of the disease and the urgent unmet medical need.

Warm autoimmune hemolytic anemia is a rare condition in which immunoglobulin G (IgG) autoantibodies bind to red blood cells, leading to their destruction. This results in anemia and can cause significant complications, including fatigue and other health challenges that impact daily life. The absence of approved targeted treatments means that patients often rely on therapies that are not specifically designed for the condition.

IMAAVY (nipocalimab-aahu) is an antibody designed to block the neonatal Fc receptor (FcRn). By inhibiting this receptor, the therapy reduces circulating IgG antibodies, including the autoantibodies responsible for red blood cell destruction. This targeted mechanism aims to address the underlying cause of the disease while potentially preserving other important immune functions, offering a more precise approach compared to traditional broad immunosuppressive treatments.

Beyond wAIHA, nipocalimab-aahu has demonstrated broad clinical potential across multiple autoimmune diseases. Positive results have been reported in Sjögren’s disease from the Phase 2 DAHLIAS trial published in The Lancet, as well as in systemic lupus erythematosus (SLE) from the Phase 2b JASMINE study. In addition, long-term data from the Phase 3 Vivacity-MG3 trial and its open-label extension have shown sustained efficacy and safety in generalized myasthenia gravis (gMG), for which the therapy has already received FDA approval. Johnson & Johnson has also submitted IMAAVY for regulatory review in wAIHA, reflecting continued momentum in expanding its potential across IgG-mediated autoimmune conditions.

If approved, IMAAVY could become the targeted treatment specifically indicated for warm autoimmune hemolytic anemia (wAIHA). This development underscores Johnson & Johnson’s commitment to addressing unmet medical needs and has the potential to significantly improve outcomes and quality of life for patients living with this challenging condition.

References

FDA grants Priority Review for IMAAVY® (nipocalimab-aahu) as the potential first approved treatment for people living with warm autoimmune hemolytic anemia (wAIHA)

Study Details | NCT04119050 | Efficacy and Safety of M281 in Adults with Warm Autoimmune Hemolytic Anemia | ClinicalTrials.gov