European Commission approves Rezurock (belumosudil) to treat chronic graft-vs-host disease – Sanofi
The European Commission has granted a conditional marketing authorisation for Rezurock (belumosudil) for the treatment of chronic graft-versus-host disease (GVHD) in adults and in children aged 12 years and older with a body weight of at least 40 kg. The medicine is to be used when other treatment options provide limited clinical benefit, are not suitable, or have been exhausted. The conditional marketing authorisation is contingent on completion of a confirmatory, randomised, controlled study. This follows the positive opinion by the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) issued on 30 January 2026.
“Chronic GVHD is a serious and potentially life-threatening condition, imposing profound physical and emotional burdens on a substantial proportion of patients following allogeneic stem cell transplantation. Across the EU, many patients continue to face significant challenges in managing this disease, particularly when existing therapies fail to provide adequate benefit,” said Mohamad Mohty, Professor of Haematology and Head of the Haematology and Cellular Therapy Department at Hôpital Saint-Antoine and Sorbonne University, Paris, France. “This approval represents an important advance, offering a new therapeutic option that has the potential to meaningfully improve the lives of patients.”
“Nearly one in two patients with chronic GVHD require third-line treatment, yet therapeutic options available for EU patients at this late stage of the disease have remained limited,” said Olivier Charmeil, Executive Vice President, General Medicines, and Interim CEO, Sanofi. “We have an ongoing commitment to supporting patients with chronic GVHD and their caregivers and are pleased to deliver this new treatment option to patients living with this debilitating and long-term condition.”
This approval is based on safety and efficacy results from several clinical studies and real-world evidence. This includes the randomised, multicenter ROCKstar phase II study (clinical study identifier: NCT03640481), which demonstrated clinically meaningful and durable responses with Rezurock in patients living with chronic GVHD after stem cell transplant and at least two prior lines of systemic therapy. Treatment was generally well tolerated. Under the conditional marketing authorisation, Sanofi will conduct a new, confirmatory, randomised, controlled study.
The medicine was designated ‘orphan’ (a medicine used in rare diseases) in 2019 for the treatment of graft-versus-host disease. Following this conditional marketing authorisation, the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency has also formally adopted an opinion on the maintenance of orphan designation. In addition to the EU, Rezurock is approved in 20 countries, including the US, UK, and Canada for the treatment of patients 12 years and older with chronic GVHD after failure of at least two prior lines of systemic therapy, and in China after failure of one prior line of systemic therapy. More than 20,000 patients living with chronic GVHD have been treated with Rezurock since its first approval in the US in July 2021.
About the ROCKstar study
ROCKstar was a pivotal phase II, open label, non-controlled, randomised, multicentre study that evaluated the efficacy and safety of Rezurock in patients with chronic GVHD after receiving two to five prior lines of systemic therapy. A three-year, open-label, follow-up analysis of the ROCKstar study evaluated the long-term efficacy of Rezurock. Treatment consisted of Rezurock 200 mg and was administered continuously until clinically significant progression of chronic GvHD or unacceptable toxicity. The primary endpoint was best overall response rate (ORR) at any time. Study results demonstrated clinically meaningful and statistically significant best ORR of 74% on treatment with Rezurock (n=77, 95% CI, 63-83 p<0.0001). The most common adverse reactions were fatigue (46%), diarrhoea (35%), nausea (35%), dyspnoea (32%), cough (30%) and upper respiratory tract infections (26%).
