HI-Bio™, a clinical-stage biotechnology company, has received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) for felzartamab, a targeted therapy for the treatment of antibody-mediated rejection (AMR) in kidney transplant recipients. This designation is a significant milestone in the development of drugs and biologics intended to treat rare diseases or conditions affecting fewer than 200,000 people in the United States.
AMR is a major cause of kidney transplant failure, with late AMR affecting approximately 23,000 patients in the U.S. Currently, there is no effective treatment for AMR, and patient options are highly limited. The development of felzartamab marks a potential breakthrough in addressing this critical unmet medical need.
Dr. Uptal Patel, Chief Medical Officer at HI-Bio, expressed optimism about the Orphan Drug Designation, stating, “Following the FDA’s granting of Breakthrough Therapy Designation for felzartamab in primary membranous nephropathy, we are encouraged to receive Orphan Drug Designation for felzartamab for antibody-mediated rejection.” He further added, “We are confident in the clinical progress of our anti-CD38 cellular depletion strategy, which has shown promising results in multiple severe immune-mediated diseases, including antibody-mediated rejection, IgA nephropathy, and primary membranous nephropathy.”
The 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. This designation is expected to accelerate the development of felzartamab and enhance the company’s ability to bring this innovative therapy to patients in need.
Felzartamab, an investigational therapeutic human monoclonal antibody directed against CD38, has demonstrated selective depletion of CD38+ plasma cells in clinical studies. This mechanism of action holds promise for improving clinical outcomes in a broad range of diseases driven by pathogenic antibodies, offering hope for patients with severe immune-mediated diseases.
The ongoing study of felzartamab in antibody-mediated rejection is anticipated to provide valuable clinical data, with the company looking forward to presenting the findings at a medical conference later this year. The progress of felzartamab in addressing AMR signifies a significant step forward in the pursuit of effective treatments for severe immune-mediated diseases, bringing renewed hope to patients and healthcare professionals alike.
