CHMP has issued a positive opinion recommending conditional EU approval of Sanofi’s Rezurock (belumosudil) for adults and adolescents with chronic GVHD after multiple prior therapies
Written By: Pharmacally Medical News Desk
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion recommending the conditional marketing authorisation of Rezurock (belumosudil) in the European Union for the treatment of chronic graft-versus-host disease (cGVHD).
The recommendation applies to adults and adolescents aged 12 years and older with a body weight of at least 40 kg, particularly for patients whose prior treatment options have provided limited benefit, are not suitable, or have been exhausted. A final European Commission decision is expected in the coming weeks.
Olivier Charmeil, Executive Vice President, General Medicines at Sanofi, noted that the company requested the re-examination of the prior negative CHMP opinion adopted in October 2025, and agreed to conduct a new post-approval confirmatory study given the limited late-line treatment options available for EU patients. Charmeil added, “We remain committed to supporting the GVHD community and welcome this positive CHMP opinion, which brings us closer to delivering a new approved treatment in the EU for adult and adolescent GVHD patients who are waiting.”
Clinical evidence and efficacy results
The CHMP recommendation is supported by safety and efficacy data from multiple clinical studies and real-world evidence, including the pivotal ROCKstar Phase 2 trial (NCT03640481). ROCKstar was a randomised, multicentre study evaluating Rezurock in patients with chronic GVHD who had received between two and five prior lines of systemic therapy following stem cell transplantation.
Patients received continuous oral Rezurock at a dose of 200 mg until clinically significant disease progression or unacceptable toxicity. The primary endpoint was best overall response rate (ORR) at any time during treatment.
Results from ROCKstar demonstrated a clinically meaningful best ORR of 74% (n=77), showing durable responses in a heavily pre-treated population. Long-term follow-up analyses extending up to three years further supported sustained benefit.
Safety profile
Rezurock was generally well tolerated in the ROCKstar study. The most frequently reported adverse reactions included fatigue (46%), diarrhoea (35%), nausea (35%), dyspnoea (32%), cough (30%), and upper respiratory tract infections (26%). As part of the conditional approval pathway, Sanofi has committed to conducting an additional confirmatory randomised controlled study to further evaluate long-term benefit and safety.
Experts highlighted the clinical significance of providing new options for patients with limited remaining therapies. Prof Mohamad Mohty, Professor of Haematology and Head of the Haematology and Cellular Therapy Department at Hôpital Saint-Antoine and Sorbonne University in Paris, stated that chronic GVHD can involve multiple organs and profoundly affect daily life, and that the positive CHMP opinion represents an important step forward for patients who have exhausted available treatment options.
Regulatory status and global approvals
Rezurock is currently approved in more than 20 countries worldwide. This includes the United States, United Kingdom, and Canada for patients aged 12 years and older with chronic GVHD after failure of at least two prior lines of systemic therapy. In China, Rezurock is approved after failure of one prior line of systemic therapy. Since its first approval in the United States in July 2021, more than 17,000 patients have been treated with Rezurock globally.
About Rezurock and its mechanism of action
Rezurock (belumosudil) is Sanofi’s first-in-class selective inhibitor of ROCK2 (Rho-associated coiled-coil kinase 2). ROCK2 is involved in immune dysregulation, inflammation, and fibrosis, which are central processes in the development and progression of chronic GVHD. By targeting this pathway, Rezurock is designed to help control inflammatory damage and reduce fibrotic complications in affected organs.
Sanofi is also investigating Rezurock in additional settings, including younger paediatric patients from one year of age and in other transplant-related complications such as chronic lung allograft dysfunction. These uses remain investigational and are not yet approved by regulatory authorities.
About chronic graft-versus-host disease
Chronic graft-versus-host disease is a potentially life-threatening complication that can occur after allogeneic hematopoietic stem cell transplantation. The condition develops when donor immune cells attack the recipient’s tissues, leading to chronic inflammation and fibrosis that can damage multiple organs.
Chronic GVHD affects up to 50% of patients who undergo allogeneic stem cell transplantation and is considered one of the leading causes of morbidity and late non-relapse mortality. Beyond physical symptoms, the disease imposes substantial emotional and socio-economic burdens, significantly impacting quality of life for patients and caregivers.
References
Press Release: Sanofi’s Rezurock recommended for EU approval by the CHMP to treat chronic graft-vs-host disease, 30 January 2026, Press Release: Sanofi’s Rezurock recommended for EU approval by the CHMP to treat chronic graft-vs-host disease
Efficacy and Safety of KD025 in Subjects With cGVHD After At Least 2 Prior Lines of Systemic Therapy, ClinicalTrials.gov ID NCT03640481, https://clinicaltrials.gov/study/NCT03640481
Cutler C et al, Belumosudil for chronic graft-versus-host disease after 2 or more prior lines of therapy: the ROCKstar Study. Blood. 2021 Dec 2;138(22):2278-2289. Erratum in: Blood. 2022 Mar 17;139(11):1772. PMID: 34265047; PMCID: PMC8641099. https://doi.org/10.1182/blood.2021012021