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FDA grants orphan drug status to BGB324 for AML

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The FDA granted orphan drug designation to BGB324 for the treatment of patients with acute myeloid leukemia, its manufacturer announced recently.

Based on preclinical research, BGB324 (BerGenBio) — a selective inhibitor of Axl receptor tyrosine kinase — has demonstrated potential in overcoming epithelial–mesenchymal transition-associated drug resistance and metastasis in carcinomas.

The FDA based its decision in part on early data from an ongoing phase 1b multicenter clinical study evaluating the safety and tolerability of BGB324, whether administered as a single agent or combined with cytarabine, in patients with AML. Secondary endpoints of this study will investigate evidence of clinical response and evaluate novel biomarkers.

“The FDA’s decision to grant BerGenBio orphan drug status for BGB324 is a significant milestone for the company and recognizes the need for innovative new ways to treat AML,” Richard Godfrey, CEO of BerGenBio, said in a press release. “The designation will give BerGenBio access to various development incentives from the Agency, including tax credits for qualified clinical testing. Additionally BerGenBio will be exempt from prescription drug user fees for BGB324 for this indication and, if the drug receives marketing approval, it will enjoy 7 years of market exclusivity in the United States.”

The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States.