The European Medicines Agency (EMA) will re-examine its October 2025 decision to reject Rezurock (belumosudil) for chronic graft-vs-host disease (cGvHD), offering a potential second chance for a therapy intended for patients with few remaining treatment options.

The Committee for Medicinal Products for Human Use (CHMP) announced at its November meeting that it would review the negative opinion after receiving a formal request from the applicant, Sanofi Winthrop Industrie.

Belumosudil is a selective inhibitor of Rho-associated coiled-coil containing protein kinase (ROCK), targeting ROCK2 and, to a lesser extent, ROCK1. In human T-cell assays (ex vivo/in vitro), it modulates immune responses by reducing STAT3/STAT5 phosphorylation and shifting the Th17/Treg balance, leading to downregulation of proinflammatory pathways. Additionally, belumosudil inhibits abnormal profibrotic signaling in vitro and shows therapeutic activity in animal models of cGvHD.

Rezurock was developed as daily tablets for treating cGvHD in adults and adolescents from 12 years of age and weighing at least 40 kg, after a blood stem cell transplant. It was to be used when other treatments had not worked well enough or were not suitable. It was designated an orphan medicine on October 17, 2019. 

Why EMA Originally Rejected It

EMA’s refusal centered on uncertainties in the benefit-risk profile, including:

• Difficulty determining the drug’s clinical effect because the main trial lacked a comparator arm and allowed concomitant treatments
• Limited robustness and durability of efficacy data
• Negative findings from an early-terminated trial evaluating Rezurock as first-line therapy, which failed to show benefit
• The company’s inability to provide additional data before 2030, despite the urgent clinical need for new cGvHD treatments 

What Happens Next

The CHMP will reevaluate the company’s arguments and render a final recommendation following the reexamination.

The company has stated that there are no consequences for patients currently enrolled in clinical trials or receiving Rezurock through compassionate use.