New medicine to treat chronic graft-versus-host disease

30 January 2026

Rezurock addresses unmet need when other treatments are unsuccessful or unsuitable

EMA has recommended granting 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.

Chronic GvHD is a long-term serious life-threatening condition, where the donor cells of a bone marrow or stem cell transplant attack the host body. The condition can affect multiple organs, leading to complications such as skin fibrosis, reduced joint mobility and lung damage, which can severely impair physical functioning and overall quality of life. Chronic GvHD affects 30 to 70% of adults and 6 to 33% of children who receive a stem cell transplant as treatment for medical conditions such as blood cancers or immunodeficiency syndromes.

Current therapies include corticosteroids, often combined with other immune-suppressing medicines. These often have limited long-term benefit and can cause significant side effects such as infections, low blood cell counts, and increased risk of new cancers. Many patients with chronic GvHD (50 to 75%) need several different lines of therapy, and effective second- and third-line treatment options are limited. In the long term, approximately one third of patients with chronic GvHD experience a relapse of their original disease or die, one third discontinue therapy successfully and one third remain on treatment. There is an unmet medical need for more effective treatment options for chronic GvHD.

The active substance in Rezurock, belumosudil, is a protein kinase inhibitor that blocks the action of ROCK2, a protein involved in the immune reactions that take place in chronic GvHD. It is given as a tablet taken once a day with food.

EMA’s recommendation is based on the results of an open-label study that included patients aged 12 years and older with chronic GvHD for whom the condition was not sufficiently controlled with corticosteroids and at least two prior lines of systemic therapy.

Over a 6-month period, 73% of patients who took Rezurock once daily had responded to treatment (with 44% still showing a response at 6 months). Around 5% of patients achieved a complete response (meaning all symptoms in all affected organs resolved) while around 68% achieved a partial response (meaning at least one organ improved and no other organ worsened or was affected). 

The most common side effects reported with Rezurock were tiredness, diarrhoea, nausea, headache, vomiting, and increased blood levels of the liver enzymes aspartate aminotransferase, alanine aminotransferase, and gamma-glutamyltransferase.

Rezurock is recommended for a conditional marketing authorisation, one of the EU regulatory mechanisms to facilitate early access to medicines that fulfil an unmet medical need. This type of approval allows the Agency to recommend a medicine for marketing authorisation with less complete data than normally expected, if the benefit of a medicine’s immediate availability to patients outweighs the risk inherent in the fact that not all the data are yet available.

In order to confirm the efficacy of Rezurock, the company has committed to carrying out a confirmatory randomised controlled study.

The opinion adopted by the CHMP is an intermediary step on Rezurock’s path to patient access. The opinion will now be sent to the European Commission for the adoption of a decision on an EU-wide marketing authorisation. Once a marketing authorisation has been granted, decisions about price and reimbursement will take place at the level of each Member State, taking into account the potential role or use of this medicine in the context of the national health system of that country.