- FDA Grants Priority Review and Sets PDUFA Date of May 30, 2021 -
- Application Being Evaluated Under FDA's Real-Time Oncology Review (RTOR) and Project Orbis Pilot Programs -
NEW YORK, NY / ACCESSWIRE / November 30, 2020 / Kadmon Holdings, Inc. (NASDAQ:KDMN) today announced that the U.S. Food and Drug Administration (FDA) has accepted the Company's New Drug Application (NDA) 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). The FDA granted Priority Review for the NDA for belumosudil, which provides for a six-month review, and assigned a Prescription Drug User Fee Act (PDUFA) target action date of May 30, 2021.
"The FDA's acceptance of our NDA for belumosudil represents an important milestone for Kadmon and further highlights the efforts of the Agency to bring meaningful new therapies to cGVHD patients as quickly as possible," said Harlan W. Waksal, M.D., President and CEO of Kadmon. "We look forward to the opportunity to bring belumosudil to market as we continue preparations for a launch, if approved."
Kadmon submitted the NDA for belumosudil under the FDA's Real-Time Oncology Review (RTOR) pilot program. This pilot program aims to explore a more efficient review process to ensure that safe and effective treatments are available to patients as early as possible, while maintaining and improving review quality.
The review of the belumosudil NDA is also being conducted under Project Orbis, an initiative of the FDA Oncology Center of Excellence. Project Orbis provides a framework for concurrent submission and review of oncology drugs among participating international partners.
The FDA previously 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 also previously granted Orphan Drug Designation to belumosudil for the treatment of cGVHD.
The NDA submission is supported by positive data from ROCKstar (KD025-213), the Company's pivotal clinical trial evaluating belumosudil in patients with cGVHD who have received two or more prior lines of systemic therapy. Twelve-month data from ROCKstar will be presented in an oral session at the 62nd American Society of Hematology (ASH) Annual Meeting on December 6th, 2020.
ROCKstar (KD025-213) is an ongoing open-label trial of belumosudil 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. Belumosudil has been well tolerated and adverse events have been consistent with those expected in the patient population.
Belumosudil (KD025) is a selective oral inhibitor of Rho-associated coiled-coil kinase 2 (ROCK2), a signaling pathway that modulates inflammatory response and pro-fibrotic processes. 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. The FDA also granted Orphan Drug Designation to belumosudil for the treatment of cGVHD. The FDA granted Priority Review for the NDA for belumosudil and assigned a PDUFA date of May, 30, 2021.
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.
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