H3 Biomedicine, a clinical stage biopharmaceutical company specializing in the discovery and development of precision medicines for oncology and a member of Eisai’s global Oncology Business Group, announced that the FDA has granted the company an orphan drug designation for H3B-6527, its first solid tumor clinical compound, for the treatment of patients with Hepatocellular Carcinoma (HCC). H3B-6527, a selective, orally bioavailable, and potent covalent inhibitor of fibroblast growth factor receptor 4 (FGFR4), is currently in phase I clinical trials.
“Receiving orphan drug designation for H3B-6527 is a significant step forward in H3’s efforts to bring a new potential treatment option to HCC patients and their families,” said Markus Warmuth, M.D., President and CEO of H3 Biomedicine. “We believe that the progress we have made thus far by targeting FGFR4 has set the stage for further clinical research, and we look forward to the continued study of H3B-6527 to determine its impact on patients living with liver cancer. We remain committed to advancing this clinical program and providing clinically meaningful benefit to patients.”
The FDA’s Office of Orphan Drug Products grants orphan status to support development of medicines for rare diseases or conditions that affect fewer than 200,000 people in the U.S. The orphan drug designation provides H3 Biomedicine with certain benefits, including market exclusivity upon regulatory approval if received, exemption of FDA application fees, and tax credits for qualified clinical trials.
This is the second clinical compound to receive Orphan Drug designation by the FDA for H3 Biomedicine. In August, 2017, the company’s lead clinical compound, H3B-8800, received orphan designation for Acute Myelogenous Leukemia (AML) and Chronic Myelomonocytic Leukemia (CMML). H3B-8800, a potent, selective and orally bioavailable small molecule modulator of wild-type and mutant SF3b complexes, is currently in phase I clinical trials.