FDA Grants Orphan Drug Designation to Kadmon's Belumosudil for the Treatment of Systemic Sclerosis
NEW YORK, NY / ACCESSWIRE / September 8, 2020 / Kadmon Holdings, Inc. (NYSE:KDMN) today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to belumosudil (KD025), the Company's Rho-associated coiled-coil kinase 2 (ROCK2) inhibitor, for the treatment of systemic sclerosis (SSc). SSc is a chronic immune disorder characterized by fibrosis of the skin and internal organs, affecting approximately 75,000 to 100,000 people in the United States.
"The dual mechanism of belumosudil, which has been shown in preclinical studies to address both the immune and fibrotic components of systemic sclerosis, represents a promising new therapeutic approach for this devastating disease for which new treatments are urgently needed," said Harlan W. Waksal, M.D., President and CEO of Kadmon. "This orphan drug designation adds momentum to our clinical development of belumosudil, which has demonstrated therapeutic potential in several immune disorders."
Belumosudil is being studied in an ongoing double-blind, placebo-controlled Phase 2 clinical trial in adults with SSc. The study is enrolling 60 patients to receive belumosudil 200 mg QD, belumosudil 200 mg BID or placebo for 24 weeks. In addition, the Company plans to initiate a small (12-15 patient), open-label Phase 2 clinical trial of belumosudil in patients with diffuse cutaneous systemic sclerosis in Q1 2021.
Belumosudil is also being developed for the treatment of chronic graft-versus-host disease (cGVHD), a complication following hematopoietic cell transplantation that leads to inflammation and fibrosis in multiple tissues. Kadmon expects to submit a New Drug Application (NDA) for belumosudil in cGVHD in the fourth quarter of 2020.
The FDA grants orphan designation to promote the development of promising products for rare conditions. It qualifies a company for certain financial benefits, including seven years of market exclusivity following marketing approval, tax credits for clinical research costs, eligibility for Orphan Product grants and the waiver of certain administrative fees.
About Belumosudil (KD025)
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 for belumosudil for the treatment of patients with cGVHD who have received at least two prior lines of systemic therapy. The FDA has also granted Orphan Drug Designation to belumosudil for the treatment of cGVHD and for the treatment of systemic sclerosis. The FDA has accepted belumosudil for the treatment of cGVHD for review under its Real-Time Oncology Review (RTOR) pilot program, which aims to explore a more efficient review process to ensure safe and effective treatments are available to patients as early as possible.
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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