The FDA approved the novel agent belumosudil (Rezurock) for adults and children with chronic graft-versus-host disease (cGVHD) that did not respond to at least two prior lines of therapy.
A first-in-class drug targeting the Rho-associated coiled-coil containing kinase 2 (ROCK2) signaling pathway, belumosudil helps modulate inflammatory response and fibrotic processes. Support for the approval came from the randomized, open-label ROCKstar trial involving about 130 patients with cGVHD treated with as many as five prior regimens. Patients were randomized to two different doses of belumosudil, and the primary endpoint was overall response rate (ORR).
Median duration of cGVHD was 25.3 months, and 48% of patients had four or more involved organs. Among patients who received the approved dose (200 mg daily), the ORR was 75% through day 1 of treatment cycle five, including complete responses in 6% of patients. In 62% of responses, patients did not require new systemic therapy for at least 12 months after response. Median time to response was 1.8 months and median duration of response was 1.9 months.
In addition to the primary endpoint, 52% of patients had clinically meaningful improvement in GVHD symptoms through day 1 of cycle seven.
“Rezurock represents a new treatment paradigm for thousands of cGVHD patients, including those with difficult-to-treat manifestations like fibrosis,” said Corey Cutler, MD, MPH, of Dana-Farber Cancer Institute in Boston, in a statement from Kadmon Holdings. “Rezurock has shown robust and durable responses across the spectrum of cGVHD and is safe and well tolerated, allowing patients to stay on therapy and achieve meaningful benefit from treatment.”
The most common (≥20% of patients) adverse events were infection, asthenia, nausea, diarrhea, dyspnea, cough, edema, hemorrhage, abdominal pain, musculoskeletal pain, headache, decreased phosphate levels, increased gamma glutamyl transferase levels, decreased lymphocytes, and hypertension.
The drug’s review and approval were carried out through FDA’s Project Orbis, which provides a framework for concurrent review of oncology drugs in the U.S., Australia, Canada, Switzerland, and England.
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