The Food and Drug Administration (FDA) has approved Rezurock (belumosudil) for the treatment of patients 12 years of age and older with chronic graft-vs-host disease (GVHD) after failure of at least 2 prior lines of systemic therapy.

Rezurock, an inhibitor of rho-associated, coiled-coil containing protein kinase, is designed to downregulate proinflammatory responses and fibrotic processes. The approval was based on data from a multicenter, open-label, randomized, phase 2 trial (ClinicalTrials.gov Identifier: NCT03640481), which evaluated the efficacy and safety of belumosudil in 65 patients with chronic GVHD who received 2 to 5 prior lines of systemic therapy. Patients received belumosudil 200mg orally once daily.

The primary endpoint was overall response rate (ORR) through cycle 7 day 1 of treatment, according to the 2014 NIH Response Criteria. The key secondary endpoint was duration of response (DOR), defined as the time from initial response to progression, death, or new systemic therapies for chronic GVHD.

Results demonstrated an ORR of 75% (95% CI, 63-85), with 6% (n=4) of patients achieving a complete response and 69% (n=45) achieving a partial response. The median DOR was 1.9 months (95% CI, 1.2-2.9) and the median time to first response was 1.8 months (95% CI, 1.0-1.9).


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Among the 49 responders, 62% (95% CI, 46-74) did not require new systemic therapy for at least 12 months following response. An exploratory analysis of patient-reported symptom “bother” showed at least a 7-point decrease in the Lee Chronic Graft-vs-Host Disease Symptom Scale summary score through cycle 7 day 1 in 52% (95% CI, 40-65) of patients.

As for safety, the most common adverse reactions observed with treatment included infections, asthenia, nausea, diarrhea, dyspnea, cough, edema, hemorrhage, abdominal pain, musculoskeletal pain, headache, decreased phosphate, increased gamma glutamyl transferase, decreased lymphocytes, and hypertension.

Rezurock is supplied as 200mg tablets in 30-count bottles and is expected to be available by late August 2021. The Company will also be offering a patient assistance program called Kadmon ASSISTTM.

References

  1. FDA approves belumosudil for chronic graft-versus-host disease. News release. US Food and Drug Administration. Accessed July 19, 2021. https://www.fda.gov/drugs/drug-approvals-and-databases/fda-approves-belumosudil-chronic-graft-versus-host-disease
  2. US FDA grants full approval of Rezurock™ (belumosudil) for the treatment of patients with chronic graft-versus-host disease (cGVHD). News release. Kadmon Pharmaceuticals, LLC. Accessed July 19, 2021. https://investors.kadmon.com/news-releases/news-release-details/us-fda-grants-full-approval-rezurocktm-belumosudil-treatment.
  3. Rezurock [package insert]. Warrendale, PA: Kadmon Pharmaceuticals, LLC; 2021.