European Commission grants conditional marketing authorization to belumosudil for cGvHD

On March 31, 2026, the European Commission (EC) granted conditional marketing authorization (CMA) to belumosudil, a selective Rho-associated coiled-coil kinase 2 (ROCK2) inhibitor, for the treatment of chronic graft-versus-host disease (cGvHD) in adults and children aged ≥12 years with a body weight of ≥40 kg. It is indicated for patients whose prior therapies have provided limited clinical benefit, are not suitable, or have been exhausted. The CMA follows a January 2026 recommendation from the European Medicines Agency (EMA) for this indication and is subject to completion of a confirmatory randomized controlled study.

This approval is supported by safety and efficacy findings from multiple clinical studies and real-world evidence, including the phase II ROCKstar study (NCT03640481), which evaluated belumosudil in patients with cGvHD after 2–5 prior lines of systemic therapy. Belumosudil 200 mg was administered continuously until clinically significant cGvHD progression or unacceptable toxicity. The best overall response rate (ORR) at any time was 74% (n = 77; 95% confidence interval [CI], 63–83; p < 0.0001). The most commonly reported adverse events (AEs) were fatigue (46%), diarrhea (35%), nausea (35%), dyspnea (32%), cough (30%), and upper respiratory tract infections (26%).

Belumosudil is the first ROCK2 inhibitor to be approved in the EU, providing a new treatment option for patients with limited alternatives.