The Food and Drug Administration (FDA)  has approved belumosudil, a kinase inhibitor, for the treatment of chronic graft-versus-host disease (cGVHD) after failure of two or more prior lines of systemic therapy.

cGVHD is a rare complication that can occur after a stem cell or bone marrow transplant. Symptoms may include skin rash, mouth sores, dry eyes, liver inflammation, development of scar tissue in the skin and joints, and damage to the lungs. The exact cause of chronic GVHD is unknown. It likely results from a complex immune-mediated interaction between the donor and recipient cells. Chronic GVHD is usually treated with prednisone or other similar anti-inflammatory or immunosuppressive medications. 

The approval of belumosudil was largely based on the results of the phase 2 KD025-213 study (NCT03640481). The study included 65 patients with cGVHD who were treated with belumosudil 200 mg taken orally once daily. The primary outcome measure was overall response rate which was 75% (6% of patients achieved a complete response; 69% achieved a partial response). The median time to first response was 1.8 months. The median duration of response, calculated from first response to progression, death, or new systemic therapies for chronic GVHD, was 1.9 months. In patients who achieved response,  62% experienced at least 12 months without death or new systemic therapy initiation.

The most common adverse reactions were infections, asthenia, nausea, diarrhea, dyspnea, cough, edema, hemorrhage, abdominal pain, musculoskeletal pain, headache, phosphate decreased, gamma glutamyl transferase increased, lymphocytes decreased, and hypertension.

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