Skip to main content

FDA Approves New Treatment Option For Chronic Graft-Versus-Host-Disease In Patients Ages 12 Years And Older

Rye Brook, N.Y., August 3, 2021 - The U.S. Food and Drug Administration (FDA) recently approved belumosudil (Rezurock™) in patients 12 years or older for the treatment chronic graft-versus-host-disease (GvHD) that has not responded or has stopped responding to earlier treatments.

GvHD is a common and serious complication of donor-derived (allogeneic) stem cell transplants, which are used to treat multiple myeloma and certain types of leukemia and lymphoma. GvHD happens when the donor’s T cells (the graft) see the patient’s healthy cells (the host) as foreign and attack them.

The acute form of the GvHD occurs in approximately 50% of stem cell transplant recipients, usually within the first 100 days after the transplant. Chronic GvHD usually appears later and can cause serious damage, including inflammation and scarring, in many tissues, including the skin, mouth, eyes, joints, liver, lung, esophagus and the GI tract. About 14,000 patients in the U.S. are living with chronic GvHD, which is one of the leading causes of medical problems and death after stem cell transplantation.

The FDA approval of once-daily oral belumosudil was based on results from the ROCKstar trial. Nearly half of the 65 patients in the trial had four or more organs affected and they had been treated with up to five drugs before beginning belumosudil. The overall response rate to the drug was 75%, with 6% of patients having a complete response and 69% having a partial response. The median time to response was 1.8 months and 62% of the patients who responded did not require a new form of therapy for at least 12 months.

Stem cell transplant offers a potential cure to patients with many types of cancer, but acute and chronic GVHD is a frequent and sometimes fatal complication. Belumosudil gives patients and healthcare providers an important new option to help with this challenging disease.