FDA Grants Orphan Drug Designation to BGB324 for Acute Myeloid Leukemia
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.
The content in this post has not been reviewed by the American Society of Clinical Oncology, Inc. (ASCO®) and does not necessarily reflect the ideas and opinions of ASCO®.
