Rare Disease Report

Therapy to Treat Transplant Complications Gets Orphan Drug Designation

OCTOBER 06, 2017
Mathew Shanley
The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to KD025, developed by Kadmon Holdings.

The drug, a Rho-associated coiled coil kinase (ROCK) inhibitor, is intended for the treatment of chronic graft-versus host disease (cGVHD), a severe complication that frequently follows allogeneic bone marrow or stem cell transplantation.

Patients with cGVHD commonly experience initial symptoms like mouth sores and raised, itchy skin rashes. The condition can cause damage to glands, resulting dry eyes,  dry mouth, and eventually causing ulcers and liver inflammation. More severe cases can present with cutaneous sclerosis (scar tissue in skin) or bronchiolitis obliterans syndrome (damage to lung passages).

The exact cause of the condition is unknown, but is likely due to complications from donor samples maturing in the patient’s body. Current treatment includes prednisone or other similar anti-inflammatory or immunosuppressive medications.

An ongoing Phase 2 clinical trial is evaluating KD025 in adults with steroid-dependent or steroid-refractory cGVHD, and 48 enrolled patients have been divided into 3 cohorts at varying dosage levels (KD025 200 mg QD, 200 mg BID and 400 mg QD).

Preliminary studies have  reported that patients receiving the lowest dose (n=17), KD025 200mg QD have demonstrated significant clinical benefit and meaningful responses.

"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 in a press release. “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.”

At the time of the designation, no drug-related serious adverse events (AEs) have been recorded in any clinical studies involving KD025.

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