Chronic graft-versus-host disease (cGVHD) is a complication that can occur after allogeneic stem cell transplantation. The transplanted immune cells (graft) attack the patient's cells (host), leading to inflammation and fibrosis in various tissues, including the skin, mouth, eyes, joints, liver, lungs, esophagus, and gastrointestinal tract. The FDA approved REZUROCK (belumosudil) for this disease, and it was quickly included in the NCCN Guidelines shortly afterward, validating REZUROCK's potential clinical impact in this difficult-to-treat area.
REZUROCK is the first and only approved therapeutic targeting Rho-associated helical kinase 2 (ROCK2), a signaling pathway that regulates inflammatory responses and fibrotic processes. The FDA granted REZUROCK Breakthrough Therapy Designation and Priority Review, and reviewed its New Drug Application (NDA) under the Real-Time Oncology Review (RTOR) pilot program.
On July 16, 2021, the U.S. Food and Drug Administration (FDA) approved REZUROCK (belumosudil) 200 mg once daily (QD) for the treatment of adult and pediatric patients aged 12 years and older with chronic graft-versus-host disease (cGVHD) who have failed at least two prior systemic therapy regimens.
The FDA approval of REZUROCK is based on the safety and efficacy results of ROCKstar (KD025-213), a randomized, open-label, multicenter pivotal trial for the treatment of cGVHD patients who have previously received 2 to 5 cycles of systemic therapy.
Based on the ROCKstar study, the NCCN guidelines recommend that "belumosudil is a promising therapy for steroid-refractory cGVHD, well-tolerated, and capable of producing clinically meaningful responses."
On August 4th, Kadmon Holdings announced that the National Comprehensive Cancer Network (NCCN) has added REZUROCK (belumosudil) tablets to the section on pre-transplant recipient assessment and graft-versus-host disease management in its NCCN Clinical Practice Guidelines for Oncology (NCCN Guidelines).
The NCCN Guidelines now list REZUROCK™ (Category 2A) as a recommended systemic treatment for steroid-refractory chronic GVHD.
Currently, belumosudil is also being developed for the treatment of systemic sclerosis, and the FDA has granted it orphan drug designation for the treatment of systemic sclerosis.
