A submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) was made for the Rho-associated coiled coil kinase 2 (ROCK2) inhibitor belumosudil (KD025) for the treatment of patients with chronic graft- versus−host disease (cGvHD). The NDA will be reviewed under the Real-Time Oncology Review pilot program. 1The FDA has previously granted belumosudil a Breakthrough Therapy designation for patients with relapsed/refractory cGvHD, which was reported on by the GvHD Hub and can be found here.
This submission comes after the results of the ROCKstar phase II trial ( NCT03640481 ), in which 132 patients, who had received ≥ 2 lines of prior therapies were treated with 200 mg of belumosudil, either once or twice daily. The primary endpoint was overall response rate. At 6 months, overall response rates of 73% and 74% were achieved in the single- and double-daily dose groups, respectively, and responses were maintained for ≥ 20 weeks in 49% of responders. No unexpected adverse events were witnessed; common severe adverse events consistied of dyspnea, GGT increase, hyperglycemia, anemia, and lung infections. The GvHD Hub reported on the positive topline results from this trial earlier this year, and these can be found here.
Belumosudil is a ROCK2-specific inhibitor that binds to and inhibits the serine/threonine kinase activity. This results in inhibition of ROCK2-mediated signaling pathways and effects the inflammatory cell responses.