Kadmon Holdings Inc. (NASDAQ:KDMN) has announced that the US FDA has accepted its New Drug Application for its Rho-associated coiled-coil kinase-2 inhibitor, belumosudil (KD025), for chronic graft-versus-host diseases treatment.
Belumosudil receives Priority Review from the FDA
Belumosudil received Priority Review from the FDA, providing a six-month review with a Prescription Drug User Fee Act (PDUFA) target action date set for May 30, 2021. Kadmon’s CEO, Harlan Waksal, said that belumosudil NDA’s acceptance is a massive milestone for the company. It further demonstrates the efforts of the FDA to deliver new therapies to cGVHD patients quickly. Harlan said that they are looking forward to the opportunity to avail belumosudil to the market as preparations continue for commercial launch once the drug receives approval.
The company had submitted the belumosudil NDA under the agency’s Real-Time Oncology Review (RTOR) pilot program. The pilot program explores a more efficient process to deliver effective and safe treatment to patients as quickly as possible while at the same time improving and maintaining review quality. Belumosudil had previously received Breakthrough Therapy Designation from the FDA for cGVHD treatment following the failure of two or more systemic therapy lines. The FDA had also granted belumosudil Orphan Drug Designation for the treatment of patients with cGVHD.
Positive ROCKstar clinical study results supported NDA submission
Kadmon supported the NDA submission with positive ROCKstar (KD025-213) clinical study data. The study evaluated belumosudil in cGVHD patients that had received two or more prior systemic therapy lines. Recently the company presented twelve-month data from the trial in an oral session on December 6, 2020, during the 62nd Annual American Society of Haematology Meeting.
The open-label ROCKstar study randomized patients into two arms of 66. Each arm was to receive belumosudil 200mg once per day and 200mg twice per day. The study’s primary endpoint was the Overall Response Rate (ORR), which the study achieved after interim analysis two months after the finalization of enrolment. Following primary analysis six months post enrolment, Belumosudil attained ORRs of 73% and 74% in the two arms, respectively.
