Belumosudil Granted Full Approval for Treatment of Chronic GVHD by FDA

The ROCK-2–targeting agent belumosudil is now approved by the FDA to treat adult and pediatric patients with chronic graft-versus-host disease after 2 prior lines of therapy.

The agent belumosudil (Rezurock) may now be used to treat adult and pediatric patients 12 years of age and older with chronic graft-versus-host disease (cGVHD) who have been unsuccessfully treated with 2 prior lines of therapy, according to the company responsible for the agent, Kadmon Holdings, Inc.1

This approval follows a priority review for the new drug application (NDA) that was granted back in November for the Rho-associated coiled-coil kinase 2 (ROCK2) inhibitor.2 The submission of the NDA was supported by data from the phase 2 ROCKstar (KD025-213) trial (NCT03640481) of belumosudil at 200 mg daily or twice daily in patients with previously treated cGVHD.

“REZUROCK represents a new treatment paradigm for thousands of cGVHD patients, including those with difficult-to-treat manifestations like fibrosis,” Corey Cutler, MD, MPH, FRCPC, Associate Professor of Medicine at Harvard Medical School and Medical Director of the Adult Stem Cell Transplantation Program at the Dana-Farber Cancer Institute, said in a press release. “REZUROCK has shown robust and durable responses across the spectrum of cGVHD and is safe and well tolerated, allowing patients to stay on therapy and achieve meaningful benefit from treatment.”

The randomized, open-label, multicenter, pivotal trial treated 65 patients at the once-daily dose, with a median time from diagnosis of 25.3 months. Of those, 48% of patients had at least 4 organs involved, 78% were refractory to their last therapy, and the median number of prior therapies was 3.

The objective response rate in the once-daily dose group was 75% (95% CI, 63%-85%) through cycle 7, day 1, comprised of 6% complete responses and 69% partial responses. The median response duration was 1.9 months.

The median time to first response was 1.8 months and 62% of patients did not require any new systemic therapy for 12 months following belumosudil treatment.

The agent appeared to be well tolerated and was consistent with the known profile of corticosteroids and/or other immunosuppressants in this setting.

“Patients receiving REZUROCK reported significant improvements in cGVHD symptoms, showing that not only did treatment result in organ responses, but it also made people feel better. This is so important for a chronic disease with a high symptom burden,” Stephanie Lee, MD, MPH, Professor at the Fred Hutchinson Cancer Research Center and the University of Washington School of Medicine and Research Director of the Long-Term Follow-Up Program at Fred Hutchinson, said in a press release.


1. U.S. FDA Grants Full Approval of REZUROCK(TM) (belumosudil) for the Treatment of Patients with Chronic Graft-Versus-Host Disease (cGVHD). Kadmon Holdings, Inc. July 16, 2021. July 16, 2021.

2. Kadmon Announces FDA Acceptance of NDA for Belumosudil in Patients With Chronic Graft-Versus-Host Disease. Kadmon Holdings, Inc. November 30, 2020. July 16, 2021.