Reimb talks for GVHD drug Rezurock extend into the New Year

Sanofi enters extended negotiations with the National Health Insurance Service

Alternative for patients failing first- and second-line therapy… reimbursement status in the new year draws attention

The graft-versus-host disease (GVHD) treatment Rezurock has stalled at the final stage on its path toward reimbursement listing in Korea.

Sanofi Korea and the National Health Insurance Service (NHIS) failed to conclude price negotiations for the ROCK2 inhibitor Rezurock (belumosudil) by the end-of-year deadline and have entered extended negotiations. As a result, reimbursement discussions have carried over into the beginning of this year.

Accordingly, it remains to be seen whether Sanofi can complete the listing process in 2026.

Rezurock, which was granted accelerated approval from the U.S. Food and Drug Administration (FDA), was approved in Korea in August 2024 and launched as a non-reimbursed product in November. Its key feature is the selective inhibition of ROCK2, a novel mechanism targeting the inflammatory response and fibrosis process in chronic graft-versus-host disease (cGVHD).

Chronic GVHD is a complication that occurs in approximately half of patients who receive allogeneic hematopoietic stem cell transplantation. While the patient population may be small due to the disease's nature, it affects half of transplant recipients and is a severe, life-threatening condition requiring essential treatment.

Graft-versus-host disease is the leading cause of death in hematologic malignancy patients, accounting for 37.8% of deaths excluding relapse. The problem is that as hematopoietic stem cell transplants increase annually in Korea (1,794 cases in 2023), treating chronic GVHD is becoming increasingly important. Among transplant patients, 42% experience chronic GVHD within an average of 3 years, and 66% have already experienced acute GVHD.

However, a significant treatment gap remains. Steroids, recommended as first-line therapy in both domestic and international treatment guidelines, are not suitable for long-term use. Prolonged steroid therapy can cause Cushing’s syndrome, which causes various systemic side effects when used for extended periods, including osteoporosis, joint necrosis, organ failure, hyperlipidemia, gastrointestinal disorders, and growth retardation.

While 96% of patients with chronic GVHD receive steroids as first-line therapy, 70% require second-line treatment, and as many as 50% ultimately need third-line therapy. When second-line therapy fails, patients are left to be treated with a combination of steroids and immunomodulators due to a lack of effective third-line options.

Furthermore, 97% of GVHD patients treated with steroids experience at least one complication, with infection (79.5%) being the most common. Systemic, multiple symptoms significantly impair patients' quality of life, and host reactions occurring in the lungs or liver are particularly fatal.

Against this backdrop, attention is focused on whether Rezurock, if granted reimbursement, can establish itself as a new treatment option.

Meanwhile, in clinical trials involving patients who had failed two or more lines of systemic therapy, Rezurock demonstrated a high overall response rate (ORR) of 75%, confirming superior efficacy over existing treatments. Notably, it showed response rates of 71%, 39%, and 26% in the joints, liver, and lungs, respectively—areas where improvement is difficult with conventional therapies.

Professor Hee Je Kim, Head of the Department of Hematology at Seoul St. Mary’s Hospital, said, “In 42% of patients with chronic graft-versus-host disease, symptoms occur at multiple sites throughout the body, significantly lowering quality of life. Host reactions occurring in the lungs and liver, in particular, can have a fatal impact on blood cancer patients, making effective management therapies urgently needed.”