The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Authority (EMA) has given a conditional marketing authorization for belumosudil (Rezurock, Sanofi) to treat chronic graft-vs-host disease (GVHD) in adults and in children aged 12 years and older with a body weight of at least 40 kg. The positive opinion was given following a re-examination after authorization was initially refused in October 2025.

GVHD is a major cause of morbidity and mortality following allogeneic hematopoietic cell transplantation. It is due to the donor T cells of a bone marrow or stem cell transplant attacking the host body in response to histo-incompatible antigens on the host tissues.

The chronic form typically develops 100 or more days post-transplant and may occur de novo or follow acute GVHD in which the early days feature various skin, gastrointestinal tract, and liver manifestations. Chronic GVHD can involve almost any organ and may resemble an autoimmune condition. It is a serious and potentially life-threatening complication. Clinical features such as skin fibrosis, reduced joint mobility, and lung damage can severely impair physical functioning and overall quality of life.

GVHD Common After Transplant 

The EMA said that chronic GVHD affects 30%-70% of adults and 6%-33% of children who receive a stem cell transplant as treatment for medical conditions such as blood cancers or immunodeficiency syndromes.

Belumosudil is a selective inhibitor of Rho protein kinase 2, which is involved in the immune reactions underlying GVHD. Blocking this protein selectively down-regulates interleukin 17 and 22 secretion and mediates signalling in immune cellular function and fibrotic pathways. The aim is both to help treat the disease and to protect the body’s organs from being attacked by the donor cells.

In a phase 2, open-label, multicenter study in patients with chronic GVHD, a response was observed in about 73% of patients treated with belumosudil plus standard of care (such as corticosteroids, calcineurin inhibitors, sirolimus, ruxolitinib, and/or extracorporeal photopheresis). The treatment yielded an overall 6 months response rate of about 44%, with a median duration of response of 23.9 weeks. 

Oral Once-Daily Dosing 

Belumosudil is available as an oral tablet taken once daily. Its most common side effects include fatigue, diarrhea, nausea, headache, and vomiting. It may increase levels of aspartate aminotransferase, alanine aminotransferase, and gamma-glutamyltransferase. Treatment should be initiated and supervised by physicians experienced in the treatment of chronic GVHD.

The CHMP said that belumosudil’s conditional marketing authorization was granted on the basis that the drug fulfils an unmet medical need when the benefit of immediate availability to public health outweighs the risk inherent in the fact that additional data are still required. It concluded that it could be be used when other treatment options provide limited clinical benefit, are not suitable, or have been exhausted. The marketing authorization holder is expected to provide comprehensive clinical data at a later stage.

Orphan Drug Status 

Belumosudil was designated an orphan medicine in 2019, and the EMA will now review the information available to date to determine if the orphan designation can be maintained.

Detailed recommendations for the use of this product will be described in the summary of product characteristics, which will be published on the EMA website in all official European Union languages after the marketing authorization has been granted by the European Commission.