What is the efficacy of Besudil mesylate tablets in patients with cGVHD who have failed multiple lines of treatment?

Belumosudil mesylate tablets (belumosudil), as an innovative kinase inhibitor, provides a new treatment option for adult and pediatric patients aged 12 years and above suffering from chronic graft-versus-host disease (cGVHD). These patients typically have experienced at least two systemic treatment failures and are at risk for severe complications such as inflammation and fibrosis. Besudil effectively reduces the inflammatory response and scarring of organ tissues that may occur in cGVHD by precisely blocking specific proteins in the immune system.

The FDA's approval of besudil was based on the safety and efficacy results of a pivotal clinical trial called ROCKstar (KD025-213). This trial is a randomized, open-label, multicenter study specifically evaluating the efficacy of besudil in patients with cGVHD who have failed two to five prior systemic therapies. In the trial, 65 patients took 200 mg of besudil orally once a day. The median time to diagnosis of cGVHD in these patients was 25.3 months, and 48% of patients had four or more organ involvement, making the condition quite complex. Patients had received a median of 3 previous systemic therapies, and 78% of patients were refractory to the last treatment.

Besudil showed encouraging efficacy data on day 1 of the 7th treatment cycleof the trial. The overall response rate (ORR) reached 75%, with 6% of patients achieving complete response and 69% achieving partial response. It is worth noting that the median time to first response was only 1.8 months, which shows the rapid onset of action of besudil. Additionally, 62% of responders did not require new systemic therapy for at least 12 months after response, further demonstrating its durable treatment effect.

In terms of safety, besudil was well tolerated, and adverse events were consistent with expectations for patients with advanced cGVHD who received corticosteroids and/or other immunosuppressants. No unexpected safety issues occurred.

In summary, Besudil Tablets provides new treatment hope for cGVHD patients who have failed multiple lines of treatment Its clinical trial data fully supports its application prospects in this refractory disease.

Reference materials:https://www.drugs.com/pro/rezurock.html