The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has issued a negative opinion on Sanofi's marketing authorization application for Rezurock (belumosudil) for the third-line treatment of adults and pediatric patients with chronic graft-versus-host disease (cGVHD). The French pharmaceutical company announced on October 17, 2025, that it will seek a re-examination of the CHMP opinion.
Regulatory Setback for Life-Threatening Condition
Chronic graft-versus-host disease is a life-threatening complication that devastates the lives of up to 50% of patients who undergo a stem cell transplant. The condition is considered one of the main causes of morbidity and late non-relapse mortality after stem cell transplant, with far-reaching consequences for patients' physical and emotional well-being as well as broader socio-economic impact.
"We are disappointed by the negative CHMP opinion for Rezurock in the EU and remain committed to serving the transplant patient community," said Olivier Charmeil, Executive Vice President, General Medicines at Sanofi. "Sanofi is confident in the body of clinical and real-world evidence that underscores Rezurock's consistent efficacy and well-established safety profile for treating third-line chronic graft-versus-host disease."
Clinical Evidence and Global Approval Status
Rezurock is supported by safety and efficacy results from several clinical studies and real-world evidence, including the randomized, multicenter ROCKstar phase 2 study. This study demonstrates consistent efficacy and tolerability for patients living with cGVHD after stem cell transplant as well as durable clinical responses over a period of three years.
Despite the European setback, Rezurock maintains a significant global presence. The drug is currently approved in 20 countries, including the US, UK and Canada for the treatment of patients 12 years and older with cGVHD after failure of at least two prior lines of systemic therapy. In China, it is approved after failure of one prior line of systemic therapy.
More than 17,000 patients living with cGVHD worldwide have been prescribed Rezurock since its first approval in the US in July 2021, demonstrating substantial real-world adoption of the therapy.
First-in-Class Mechanism of Action
Rezurock (belumosudil) is a first-in-class selective ROCK2 (Rho-associated coiled-coil kinase 2) inhibitor. The drug represents a novel therapeutic approach for addressing chronic graft-versus-host disease, a complication that occurs following stem cell transplant where the donor's cells attack the host's cells, leading to inflammation and fibrosis that can damage multiple tissues and organs.
Ongoing Development Programs
Sanofi remains committed to investigating the benefits of Rezurock in other age groups and indications. The company has ongoing studies for pediatric patients with cGVHD from one year old who have been treated with at least two prior lines of systemic therapy, as well as studies for patients with chronic lung allograft dysfunction.
The company expects a final decision from European regulators in the coming months following the re-examination process. Sanofi will continue to work closely with the European Medicines Agency with the aim of bringing this treatment to patients in the EU who are waiting for therapeutic options.
