FDA Grants Orphan Drug Designation to KD025 for cGVHD

Kadmon announced the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to KD025, the company’s Rho-associated coiled-coil kinase (ROCK) inhibitor, for the treatment of chronic graft-versus-host disease (cGVHD), 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 (KD025 200 mg QD, 200 mg BID and 400 mg QD), enrolled sequentially following a safety assessment of each cohort. As previously reported, in a preliminary analysis of data from the lowest-dose cohort (n=17), KD025 200 mg QD demonstrated clinically meaningful responses, with no drug-related serious adverse events recorded.

"cGVHD is a debilitating condition with few available therapies, and many patients lack treatment options," said Harlan W. Waksal, M.D., President and CEO at Kadmon. "We are pleased with the continued progress of KD025 in the ongoing clinical trial, and we anticipate presenting additional data from the study toward the end of this year."

The FDA grants orphan designation to promote the development of promising products for rare conditions affecting fewer than 200,000 U.S. patients annually. It qualifies a company for certain financial benefits, including seven years of market exclusivity following marketing approval, tax credits for clinical research costs, eligibility for Orphan Product grants and the waiver of certain administrative fees.