Application Being Reviewed Under FDA's Real-Time Oncology Review (RTOR) Pilot Program
NEW YORK, NY / ACCESSWIRE / September 30, 2020 / Kadmon Holdings, Inc. (NYSE:KDMN) today announced the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for belumosudil (KD025), the Company's Rho-associated coiled-coil kinase 2 (ROCK2) inhibitor, for the treatment of patients with chronic graft-versus-host disease (cGVHD).
"Today's NDA submission under the FDA's Real-Time Oncology Review pilot program marks an exciting milestone for Kadmon and for patients living with cGVHD," said Harlan W. Waksal, M.D., President and CEO of Kadmon. "We believe the robust and durable clinical trial results achieved with belumosudil demonstrate its potential to offer meaningful clinical benefit to cGVHD patients. We look forward to our continued dialogue with the FDA as we continue to make preparations for potential launch, if approved."
As previously reported, the NDA is being reviewed under the Real-Time Oncology Review (RTOR) pilot program, an initiative of the FDA's Oncology Center of Excellence. The RTOR program aims to explore a more efficient review process to ensure that safe and effective treatments are available to patients as early as possible.
The NDA submission is supported by positive data from ROCKstar (KD025-213), the Company's pivotal clinical trial evaluating belumosudil in 132 patients with cGVHD who have received two or more prior lines of systemic therapy. As previously reported, belumosudil achieved clinically meaningful and statistically significant Overall Response Rates (ORR) of 73% with 200 mg once daily and 74% with 200 mg twice daily. Responses were achieved across key patient subgroups and complete responses were observed in all organ systems. Belumosudil has been well tolerated and adverse events have been consistent with those expected in the patient population. Data from ROCKstar are expected to be presented at an upcoming medical meeting.
In October 2018, the FDA granted Breakthrough Therapy Designation to belumosudil for the treatment of patients with cGVHD after failure of two or more lines of systemic therapy.
ROCKstar (KD025-213) is an ongoing open-label trial of belumosudil (KD025) in patients with cGVHD who have received at least two prior lines of systemic therapy. Patients were randomized to receive belumosudil 200 mg once daily or 200 mg twice daily, enrolling 66 patients per arm. The primary endpoint of the study is Overall Response Rate (ORR). The ORR endpoint was met at the interim analysis, conducted two months after completion of enrollment. At the study's primary analysis, conducted six months after completion of enrollment, belumosudil achieved ORRs of 73% and 74% in the respective arms. Data from ROCKstar are expected to be presented at an upcoming medical meeting.
Belumosudil (KD025) is a selective oral inhibitor of Rho-associated coiled-coil kinase 2 (ROCK2), a signaling pathway that modulates inflammatory response. The FDA has granted Breakthrough Therapy Designation to belumosudil for the treatment of patients with cGVHD after failure of two or more lines of systemic therapy. The FDA has also granted Orphan Drug Designation to belumosudil for the treatment of cGVHD. The belumosudil NDA is being reviewed under the FDA's Real-Time Oncology Review (RTOR) pilot program. Belumosudil is also being studied in an ongoing Phase 2 clinical trial in adults with diffuse cutaneous systemic sclerosis (KD025-209).
cGVHD is a common and often fatal complication following hematopoietic stem cell transplantation. In cGVHD, transplanted immune cells (graft) attack the patient's cells (host), leading to inflammation and fibrosis in multiple tissues, including skin, mouth, eye, joints, liver, lung, esophagus and gastrointestinal tract. Approximately 14,000 patients in the United States are currently living with cGVHD.
Kadmon is a clinical-stage biopharmaceutical company that discovers, develops and delivers transformative therapies for unmet medical needs. Our clinical pipeline includes treatments for immune and fibrotic diseases as well as immuno-oncology therapies.
Forward Looking Statements
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Ellen Cavaleri, Investor Relations
SOURCE: Kadmon Holdings, Inc.