The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to BGB324 for the treatment of acute myeloid leukemia (AML). BGB324 is a first-in-class, highly selective small-molecule inhibitor of the Axl receptor tyrosine kinase. It blocks the epithelial-mesenchymal transition (EMT), which is a key driver in drug-resistance and metastasis.
Earlier this month BerGenBio announced that the first patient has been dosed in its multicenter phase Ib trial of BGB324 in patients with AML. The two-part trial will primarily investigate the safety and tolerability of BGB324 when administered as a single agent and in combination with a standard-of-care drug (cytarabine). Secondary endpoints will also explore evidence of clinical response and assess novel biomarkers.
The FDA grants Orphan Drug designation to development-stage novel therapeutics that are intended for use in treating rare diseases and medical conditions that affect fewer than 200,000 patients in the United States. ■