The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion recommending conditional marketing authorisation for Rezurock (belumosudil) in the European Union, marking a significant step forward for patients living with chronic graft-versus-host disease (GVHD).
If approved by the European Commission, Rezurock would be indicated for adults and adolescents aged 12 years and older who weigh at least 40 kg and are living with chronic GVHD after stem cell transplantation. The treatment is intended for patients whose disease has not adequately responded to available therapies, where other options offer limited clinical benefit, are unsuitable, or have already been exhausted. A final decision from the European Commission is expected in the coming weeks.
The positive recommendation follows a re-examination requested by Sanofi after the CHMP initially issued a negative opinion in October 2025. The reassessment considered additional clinical evidence and Sanofi’s commitment to conduct a post-approval confirmatory study, a key requirement under conditional marketing authorisation.
Chronic GVHD is a serious and potentially debilitating condition that can affect multiple organs, including the skin, liver, lungs, eyes, and gastrointestinal tract. It often leads to long-term complications that significantly impair quality of life. Commenting on the development, Prof. Mohamad Mohty, Professor of Haematology and Head of the Haematology and Cellular Therapy Department at Hôpital Saint-Antoine and Sorbonne University in Paris, said the decision represents meaningful progress for patients with limited options. He noted that for individuals who have exhausted existing treatments, the CHMP’s opinion offers renewed hope in managing a complex and burdensome disease.
Sanofi highlighted the unmet medical need in this patient population and the growing body of evidence supporting Rezurock’s use. Olivier Charmeil, Executive Vice President of General Medicines at Sanofi, said the company sought re-examination given the scarcity of late-line treatments in Europe and the inclusion of European patient data in Rezurock’s clinical development. He added that the company remains committed to supporting the GVHD community and advancing access to new therapies.
The CHMP’s recommendation is supported by data from multiple clinical trials and real-world studies, including the pivotal randomised, multicentre ROCKstar Phase II study. Results from this trial showed clinically meaningful and durable responses in patients with chronic GVHD who had received at least two prior lines of systemic therapy following stem cell transplantation. Rezurock was generally well tolerated across studies.
Rezurock is already approved in around 20 countries, including the United States, the United Kingdom, and Canada, for patients aged 12 years and older with chronic GVHD after failure of at least two prior systemic therapies. In China, it is approved after failure of one prior line of therapy. Since its first approval in the US in July 2021, more than 17,000 patients worldwide have been treated with Rezurock, underscoring its established role in addressing an area of high unmet need.