FDA approves Niktimvo for chronic GVHD
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The FDA approved axatilimab-csfr for certain patients with chronic graft-versus-host disease.
The indication applies to use of the therapy by adults and children who failed at least two prior lines of systemic therapy and who weigh at least 40 kg.
Axatilimab-csfr (Niktimvo; Incyte Corporation, Syndax Pharmaceuticals) is a colony stimulating factor-1 receptor-blocking antibody.
The FDA based approval on results of the randomized phase 2 AGAVE-201 trial, which included patients with recurrent or refractory chronic GVHD who required additional treatment after at least two lines of systemic therapy.
Researchers examined three doses of IV axatilimab-csfr — 0.3 mg/kg every 2 weeks, 1 mg/kg every 2 weeks or 3 mg/kg every 4 weeks.
Overall response rate through day 1 of the seventh treatment cycle served as the major efficacy outcome.
Researchers reported an ORR of 75% (95% CI, 64-84) among the 79 patients treated at the now-recommended dose of 0.3 mg/kg every 2 weeks.
Results showed median time to first response of 1.5 months (range, 0.9-5.1) and median response duration of 1.9 months (95% CI, 1.6-3.5).
Among patients who responded to the recommended dose, 60% (95% CI, 43-74) remained alive with no new systemic therapy initiation for at least 12 months.
The most common adverse events reported with axatilimab-csfr included increases in aspartate aminotransferase, alanine aminotransferase, gamma glutamyl transferase, amylase, calcium, creatine phosphokinase, alkaline phosphatase and lipase; decreases in phosphate and hemoglobin; infection; musculoskeletal pain; fatigue; headache; nausea; cough; diarrhea; pyrexia and dyspnea.
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