Kadmon receives orphan drug designation for KD025 for treatment of cGVHD
Kadmon announced that the U.S. Food and Drug Administration has granted orphan drug designation to KD025, the company's Rho-associated coiled-coil kinase inhibitor, for the treatment of chronic graft-versus-host disease, a serious complication following allogeneic bone marrow or stem cell transplantation. KD025 is being studied in an ongoing Phase 2 clinical trial in adults with steroid-dependent or steroid-refractory cGVHD and active disease. The dose-finding study includes 48 patients divided into three cohorts at different dose levels, enrolled sequentially following a safety assessment of each cohort. As previously reported, in a preliminary analysis of data from the lowest-dose cohort, KD025 200 mg QD demonstrated clinically meaningful responses, with no drug-related serious adverse events recorded.