Pharma Focus America

HI-Bio Receives FDA Orphan Drug Designation for Felzartamab for the Treatment of Membranous Nephropathy

Friday, May 26, 2023

Human Immunology Biosciences (HI-Bio™), a clinical-stage biotechnology company developing targeted therapies for patients with severe immune-mediated diseases (IMDs), today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) for felzartamab, the company's investigational agent that is in development for the treatment of primary membranous nephropathy (PMN). Felzartamab is an investigational monoclonal antibody designed to deplete CD38+ plasma cells, which are believed to drive PMN through the production of pathogenic autoantibodies. The company intends to advance felzartamab into late-stage studies.

"The FDA's orphan drug designation for felzartamab is an acknowledgment of the critical and ongoing unmet need in PMN and the potential for felzartamab to be an important new therapeutic option to improve the lives of patients with PMN who currently have very limited treatment options," said Uptal Patel, M.D., Chief Medical Officer at HI-Bio. "We are excited to advance our clinical development program to make progress on our goal of transforming PMN into a well-managed condition."

PMN is a rare IMD affecting the kidneys, impacting more than 36,000 people in the United States. There are currently no approved therapies for PMN. Standard of care comprises off-label use of a variety of agents, including immunosuppressive therapies like cyclophosphamide, and CD20-targeted B-cell depleting agents. Even with these strategies, half of patients with PMN continue to have nephrotic syndrome and nearly one third progress to kidney failure.

As recently announced, positive data was achieved for felzartamab in patients with PMN across two Phase 2 studies. Beyond PMN, felzartamab is currently in development for IgA Nephropathy and Antibody Mediated Rejection of kidney transplants. The company intends to explore developing felzartamab in additional IMDs where patients have serious unmet needs.

The FDA's Orphan Drug Designation program is designed to advance the development of drugs and biologics intended to treat a rare disease or condition that affects fewer than 200,000 people in the United States. Orphan drug designation qualifies HI-Bio for certain development incentives, including tax credits for qualified clinical trials, exemption of FDA application fees and up to seven-year market exclusivity upon regulatory approval.

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