Healthcare products company Johnson & Johnson (NYSE:JNJ) announced on Monday that the US Food and Drug Administration (FDA) has approved IMAAVY (nipocalimab-aahu) for the treatment of warm autoimmune haemolytic anaemia (wAIHA), a rare, life-threatening autoantibody disease, in adults and paediatric patients 12 years of age and older currently or previously treated with corticosteroids.
The company says that this approval, which follows FDA Priority Review, marks the first time a therapy has been proven safe and effective specifically for the treatment of wAIHA.
In wAIHA, pathogenic immunoglobulin G (IgG) autoantibodies attach to and destroy red blood cells, causing severe anaemia, profound fatigue, and a significantly increased risk of morbidity and mortality. Previously, available treatment options only included corticosteroids and immunosuppressants -- therapies that suppress the entire immune system without specifically targeting the IgG autoantibodies driving the disease.
The primary endpoint of the Phase 2/3 ENERGY study was durable haemoglobin (Hgb) response, reflecting meaningful increases in Hgb levels over time.
According to the company, the randomised, placebo-controlled trial demonstrated that approximately three times as many patients receiving the approved dose of IMAAVYg achieved durable Hgb levels versus placebo by 24 weeks. Overall patients in this treatment group showed a mean increase in Hgb of 1g/dL at Week 1.5. In addition, IMAAVY was associated with a 3.5-point higher mean FACIT-Fatigue score versus placebo at Week 24, with higher scores defined as less fatigue. IMAAVY demonstrated a safety profile consistent with its established safety profile in generalised myasthenia gravis (gMG).
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