August 2021: After failure of at least two prior lines of systemic therapy, the Food and Drug Administration approved belumosudil (Rezurock, Kadmon Pharmaceuticals, LLC), a kinase inhibitor, for adult and paediatric patients 12 years and older with chronic graft-versus-host disease (chronic GVHD).
KD025-213 (NCT03640481), a randomised, open-label, multicenter dose-ranging experiment in which 65 patients with chronic GVHD were treated with belumosudil 200 mg administered orally once day, was used to assess efficacy.
The overall response rate (ORR) through Cycle 7 Day 1 was the primary efficacy end measure, with overall response defined as a full response (CR) or partial response (PR) according to the 2014 NIH Consensus Development Project on Clinical Trials in Chronic Graft-versus-Host Disease guidelines. The ORR was 75% (95 percent CI: 63, 85); 6% of patients had a complete response, and 69 percent had a partial response. The average time it took to get a first answer was 1.8 months (95 percent CI: 1.0, 1.9). The median duration of response for chronic GVHD was 1.9 months, measured from first response through progression, death, or new systemic treatments (95 percent CI: 1.2, 2.9). No mortality or new systemic medication initiation occurred in 62 percent (95 percent CI: 46, 74) of patients who achieved response for at least 12 months after response.
Infections, asthenia, nausea, diarrhoea, dyspnea, cough, edoema, haemorrhage, abdominal pain, musculoskeletal pain, headache, phosphate decreased, gamma glutamyl transferase increased, lymphocytes decreased, and hypertension were the most common adverse reactions (20%), including laboratory abnormalities.
Belumosudil should be taken once a day, with food, in a dose of 200 mg.