BVX001 granted Orphan Drug Designation by the U.S. FDA

On April 17, 2024, the U.S. Food and Drug Administration granted Orphan Drug Designation to BVX001, a novel twin CD33/CD7 targeting bispecific antibody-drug conjugate, for the treatment of patients with acute myeloid leukemia (AML).¹ BVX001 selectively targets CD7+/CD33+ cells, which are present in 15–30% of patients with AML and are rarely detected in normal white blood cells.²

Preclinical data has previously demonstrated that BVX001 was well tolerated, with a favorable safety profile when compared with gemtuzumab ozogamicin in a CD34-boosted humanized murine model.³ The number of healthy neutrophils and leukocytes present were significantly lower with gemtuzumab ozogamicin when compared with the equivalent dose of BVX001 at 14 days post-injection.³ Additionally, in a preclinical AML murine model study, the median survival for BVX001-treated mice was 129 days vs 91 days for high-dose cytarabine-treated mice and 57 days for untreated controls.⁴

An Initial Targeted Engagement for Regulatory Advice (INTERACT) meeting with the U.S. Food and Drug Administration Center for Drug Evaluation and Research was also conducted, in preparation for an Investigational New Drug application for BXV001.¹