FDA Approves Belumosudil for Chronic Graft-Versus-Host Disease

Graft-versus-host disease.

The FDA has granted approval to belumosudil (Rezurock™, Kadmon Pharmaceuticals, LLC), for adult and pediatric patients 12 years and older with chronic graft-versus-host disease (GVHD) after failure of at least two prior lines of systemic therapy.

“Belumosudil (KD025) is a novel oral selective Rho-associated coiled-coil kinase 2 (ROCK2) inhibitor specifically designed for the treatment of chronic GVHD,” wrote Corey S. Cutler, MD, MPH, FRCPC, Medical Director of the Adult Stem Cell Transplantation Program at the Dana-Farber Cancer Institute and Associate Professor of Medicine at Harvard Medical School, and colleagues, in their published abstract of the results of the phase 2 ROCKstar study (NCT03640481), on which the approval was based. “In a previous dose-finding study (KD025-208, N=54), two-thirds of patients, including those with fibrotic and inflammatory manifestations, achieved a partial or complete response with belumosudil.”

In this open-label, randomized, multicenter study, eligible patients had chronic GVHD and had received two to five prior lines of therapy. Out of 132 enrolled patients, 66 patients received belumosudil 200 mg once a day. The other arm, which consisted of 66 patients, received belumosudil 200 mg twice daily. The primary end point was overall response rate through Day 1 of Cycle 7. Other end points included duration of response, Lee Symptom Scale score, failure-free survival, corticosteroid dose reductions, and overall survival.

The FDA reports that the overall response rate was 75% among patients receiving the once-daily dose; 6% of patients achieved a complete response. The median duration of response, which included the time from first response to progression, death, or initiation of a new systemic therapy for GVHD, was 1.9 months. Among patients who achieved a response, no death or new systemic therapy initiation occurred in 62% of patients for at least 12 months following the response.

The most common adverse events and laboratory abnormalities occurring in at least 20% of patients were infections, asthenia, nausea, diarrhea, dyspnea, cough, edema, hemorrhage, abdominal pain, musculoskeletal pain, headache, phosphate decreased, gamma glutamyl transferase increased, lymphocytes decreased, and hypertension.

“Responses were durable and clinically meaningful, irrespective of patient and chronic GVHD characteristics,” concluded Dr. Cutler and colleagues in their abstract presented at the December 2020 American Society of Hematology (ASH) Annual Meeting and Exposition. “Belumosudil was well tolerated, with limited and manageable adverse events. Further studies will evaluate its use earlier in disease management.”

The recommended dose of belumosudil is 200 mg, taken orally once daily with food.

For More Information

Cutler C, Lee SJ, Arai S, et al (2020). Belumosudil for chronic graft-versus-host disease (cGVHD) after 2 or more prior lines of therapy: the Rockstar study (KD025-213). Blood (ASH Annual Meeting Abstracts). Abstract 353.

Clinicaltrials.gov (2021). Efficacy and safety of KD025 in subjects with cGVHD after at least 2 prior lines of systemic therapy. NLM identifier: NCT03640481.

Rezurock™ (belumosudil) prescribing information (2021). Kadmon Pharmaceuticals, LLC. Available at: https://www.accessdata.fda.gov/drugsatfda_docs/label/2021/214783s000lbl.pdf

US Food & Drug Administration (2021). FDA approves belumosudil for chronic graft-versus-host disease. Available at: https://www.fda.gov/drugs/drug-approvals-and-databases/fda-approves-belumosudil-chronic-graft-versus-host-disease

Image credit: Mark ong. Licensed under CC BY-SA 4.0

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