Sanofi’s Rezurock marks turning point in treating blood cancer complication, GVHD
A new drug has emerged that could create a turning point in the treatment of graft-versus-host disease (GVHD), a serious complication that can occur after treatment for blood cancers.
Sanofi's Rezurock (belumosudil mesylate) has surfaced as a new hope for patients with chronic GVHD, and calls are growing for improving the treatment environment in Korea.
Graft-versus-host disease is a complication that can occur in patients with blood cancer who have received an allogeneic hematopoietic stem cell transplant. It is a severe disease that causes a host response that causes several life-threatening symptoms throughout the body.
As the transplanted cells adapt to the recipient body, i.e., the host, an immunologic response is triggered, leading to inflammation, fibrosis, and other systemic damage. Graft-versus-host disease is the leading cause of non-relapsing mortality in patients with hematologic cancers, accounting for 37.8 percent of all non-relapsing deaths.
Treatment of this disease has been limited to conventional steroids and JAK inhibitors, and many patients fail first- and second-line therapy and are faced with the reality that there is no longer appropriate treatment. Existing treatments have shown low response rates in major fibrotic organs, including the lungs and joints, and patients often have inadequate response rates.
“Chronic graft-versus-host disease is a ticking time bomb that patients don't know when it will go off, as it manifests itself simultaneously throughout the entire body, starting with the oral mucosa and later on the skin, eyes, muscles, tendons, heart, and immune system,” said Professor Park Yong of the Department of Medical Oncology and Hematology at the Korea University Anam Hospital.
Professor Park explained that fibrosis irreversibly changes the tissue structure and stiffness increases. This can lead to permanent damage to organs, such as stiffened joints that make it difficult to move around or hardened lungs that make it harder to breathe, which can lead to problems with basic life functions.
Notably, for organs that have been irreversibly damaged by fibrosis, currently, available treatments are ineffective, often providing only a symptomatic rather than curative treatment, creating a high unmet clinical need, Park added.
Rezurock is the first treatment to selectively inhibit the ROCK2 signaling pathway, which is responsible for inflammation and fibrosis in chronic graft-versus-host disease.
In clinical trials in patients who have failed two or more systemic therapies, Rezurock demonstrated a high overall response rate (ORR) of 75 percent, demonstrating superior effectiveness compared to existing therapies. In particular, the response rate was 71 percent, 39 percent, and 26 percent in the joints, liver, and lung, respectively, which are difficult to improve with existing therapies, showing promising results.
“Clinicians and patients are excited about Rezurock because it was developed to target chronic graft-versus-host disease to control both inflammation and fibrosis, and the clinical data shows that it is expected to improve host responses in areas such as the lungs, liver, joints, and eyes, where existing therapies have been inadequate,” Professor Park said. “The clinical interest in Rezurock is growing as we see cases of fibrosis-induced sclerosis being alleviated with the treatment.”
Park emphasized that graft-versus-host disease is associated with an increased risk of death and irreversible tissue damage the longer the disease progresses, so early treatment is critical. Despite the current lack of an appropriate treatment after second-line therapy, many patients in Korea are not eligible for health insurance coverage, so government support through reimbursement is urgently needed to ensure that patients do not miss out on treatment opportunities.
Last October, Sanofi announced its plans to apply for reimbursement at a news conference to celebrate Rezulock’s approval in Korea. Industry insiders are interested in seeing if this year marks a turning point in the country's treatment landscape for chronic graft-versus-host disease patients.