Production & Manufacturing
Process & Production

Kadmon’s cGVHD drug gets FDA orphan drug designation

PBR Staff Writer Published 09 October 2017

Kadmon has secured orphan drug designation for its Rho-associated coiled-coil kinase (ROCK) inhibitor dubbed as KD025 from the US Food and Drug Administration (FDA) for treating chronic graft-versus-host disease (cGVHD).

The FDA’s orphan designation is granted to encourage the development of potential products for rare conditions that affect less than 200,000 patients annually in the US.

cGVHD is a serious complication that can occur after transplantation of allogeneic bone marrow or stem cell.

Currently, KD025 is undergoing a phase 2 clinical trial featuring 48 adult patients with steroid-dependent or steroid-refractory cGVHD and active disease. The dose-escalation trial will assess the safety, tolerability and activity of Kadmon’s ROCK inhibiting drug among the subjects.

Patients enrolled in the trial have been divided into three groups and are subjected to different dose levels KD025 200mg QD, 200mg BID and 400mg QD after a safety assessment of each cohort.

In the first group, patients are daily given two 100mg capsules of KD025. In the second group, patients are given two 100mg capsules of KD025 daily twice.

The third group will see four 100mg capsules of KD025 given daily once to the patients.

The phase 2 trial, which began in August 2016 across multiple centers in the US, is scheduled to end in August 2018.

Kadmon president and CEO Harlan W. Waksal said: “cGVHD is a debilitating condition with few available therapies, and many patients lack treatment options.

“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 orphan designation qualifies Kadmon for certain financial benefits from the FDA should KD025 be approved for cGVHD. These include seven years of market exclusivity, tax credits for costs spent on clinical research among others.