*The European Medicines Agency (EMA) has recommended granting a conditional marketing authorization for the sale of Rezurock (belumosudil), developed by the French company Sanofi, for the treatment of chronic graft-versus-host disease (cGVHD) in adults and pediatric patients aged 12 years and older weighing at least 40 kg. The drug is indicated for use in cases where other treatment options have proven ineffective or have been exhausted.*
GVHD is a life-threatening condition in which donor bone marrow or stem cells attack the recipient’s body. The condition can affect multiple organs, leading to fibrosis, reduced joint mobility, and lung damage. This reaction occurs in 30–70% of adults and 6–33% of children who have undergone stem cell transplantation for cancer or immunodeficiency syndromes. Belumosudil, a protein kinase inhibitor, blocks the ROCK2 protein involved in the immune response. The drug is administered orally once daily.
Corticosteroids, typically in combination with other immunosuppressants, are commonly used to treat the disease. However, such therapy has limited long-term efficacy and is associated with serious adverse effects. “There is an unmet medical need for more effective treatment options for chronic graft-versus-host disease,” the regulator noted in its statement.
The EMA recommendation is based on results from an open-label clinical trial involving patients who had failed to respond to corticosteroids and at least two prior lines of systemic therapy. Within six months of the trial, 73% of subjects responded to Rezurock treatment, with 44% maintaining response after treatment completion. Approximately 5% achieved complete remission, while 68% achieved partial remission.
Sanofi acquired the rights to Rezurock in 2021 through its $1.9 billion acquisition of Kadmon, the drug’s original developer. The deal was finalized less than two months after the U.S. Food and Drug Administration (FDA) approved the drug as a third-line therapy for chronic GVHD. In Russia, Rezurock is procured by the “Circle of Kindness” foundation for the treatment of children with life-threatening conditions.
Conditional marketing authorization — a mechanism facilitating patient access to therapies addressing unmet medical needs. Authorization is granted based on less comprehensive data than normally required, provided such data indicate that the benefits of the drug outweigh the risks and that the applicant is able to supply comprehensive clinical data at a future date.