Belumosudil Earns Scottish Approval in Chronic Graft-Versus-Host-Disease

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Data from the phase 2 ROCKstar trial support the approval of belumosudil as a treatment for patients with chronic graft-versus-host-disease in Scotland.

The FDA previously approved belumosudil for patients with chronic GVHD in July 2021.

The FDA previously approved belumosudil for patients with chronic GVHD in July 2021.

The Scottish Medicines Consortium has approved belumosudil (Rezurock) as a suitable treatment option for patients 12 years and older with chronic graft-versus-host disease (GVHD) previously treated with a minimum of 2 prior lines of therapy, according to a press release from Sanofi.1

Supporting data for belumosudil’s acceptance in Scotland came from the phase 2 ROCKstar trial (NCT03640481), in which investigators evaluated the agent among 132 patients with chronic GVHD. Overall, 75% of patients had a response at 12 months of treatment, which included responses in organs such as the lungs, liver, and skin. In the modified intent-to-treat population (n = 126), 7 patients experienced a complete response in all affected organs.

Frequent adverse effects (AEs) following treatment with belumosudil in the ROCKstar trial included fatigue (38%), diarrhea (33%), nausea (31%), cough (28%), and upper respiratory tract infection (27%). Additionally, 38% of the population experienced serious AEs, with patients most frequently experiencing pneumonia (8%). Overall, the safety profile of belumosudil in the ROCKstar trial was comparable with those of corticosteroids and other immunosuppressant agents.

“Donor stem cell transplant successfully treats thousands of people with blood cancers, but rejection of the patient by the donor transplant cells –[chronic] GVHD–is a significant clinical challenge,” Anne Parker, MD, MB, ChB, consultant hematologist at Queen Elizabeth University Hospital, NHS Greater Glasgow and Clyde, said in the press release. “It can cause damage to any body system which can be life changing and potentially life threatening. Until now there have been only a few treatment options available for these patients. I’m very pleased that belumosudil is now available and can potentially help patients who [have not responded] to current therapies.”

The FDA previously approved belumosudil for patients with chronic GVHD in July 2021.2 Findings from the ROCKstar trial supported the regulatory agency’s approval.

“[Belumosudil] represents a new treatment paradigm for thousands of patients [with chronic GVHD], including those with difficult-to-treat manifestations like fibrosis,” Corey S. Cutler, MD, MPH, FRCPC; director of Clinical Research, Stem Cell Transplantation; director of the Stem Cell Transplantation Survivorship Program; and an institute physician at Dana-Farber Cancer Institute, as well as an associate professor of Medicine at Harvard Medical School, said in a press release at the time of the FDA approval.

References

  1. Sanofi’s Rezurock® (belumosudil) accepted in Scotland for the treatment of chronic graft-versus-host disease. News release. Sanofi. July 10, 2023. Accessed July 11, 2023.
  2. U.S. FDA grants full approval of REZUROCK(TM) (belumosudil) for the treatment of patients with chronic graft-versus-host disease (cGVHD). News release. Kadmon Holdings, Inc. July 16, 2021. Accessed July 11, 2023. https://shorturl.at/pwM37
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