Johnson & Johnson has secured a significant regulatory win, with the FDA approving its therapy Imaavy for a second indication targeting a rare and life-threatening blood condition.
The approval covers Imaavy, known generically as nipocalimab-aahu, as the first treatment specifically indicated for warm autoimmune hemolytic anemia, referred to as wAIHA.
The condition causes the immune system to mistakenly destroy red blood cells, leading to severe anemia, fatigue, blood clots, and kidney failure in affected patients.
The FDA granted approval following Priority Review, extending the drug’s use to adults and paediatric patients aged 12 years and older who are currently or previously treated with corticosteroids.
The regulatory decision was supported by data from the Phase 2/3 randomised, placebo-controlled ENERGY trial, which examined the drug’s efficacy over a 24-week period.
In that trial, approximately three times as many patients receiving the approved 30 mg/kg intravenous dose every four weeks achieved a durable haemoglobin response compared to those on placebo by Week 24.
Imaavy-treated patients also showed a mean haemoglobin increase of 1 g/dL at Week 1 and a 3.5-point higher mean FACIT-Fatigue score at Week 24 versus placebo.
The drug works by blocking a protein that keeps harmful antibodies circulating in the bloodstream, lowering their levels while preserving other immune functions, and is administered intravenously every four weeks at a weight-based dose.
Johnson & Johnson estimates that approximately one in 8,000 people have wAIHA, with one to three new cases diagnosed annually per 100,000 people globally.
Imaavy was originally approved in April 2025 to treat generalised myasthenia gravis in certain adults and patients aged 12 and older, a rare immune disorder that causes progressive muscle weakness.
The latest approval marks J&J’s first label expansion for the therapy, and the company is now pursuing broader ambitions for the monoclonal antibody across a range of other indications.
J&J has advanced Imaavy to Phase 3 trials in chronic inflammatory demyelinating polyneuropathy and to Phase 2 in both systemic lupus erythematosus and idiopathic inflammatory myopathy.
The expanding clinical pipeline signals J&J’s (JNJ) confidence that nipocalimab-aahu could become a cornerstone therapy across multiple rare autoantibody-driven diseases in coming years.