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The Food and Drug Administration has granted orphan drug designation to BGB324 for the treatment of patients with acute myeloid leukemia (AML). BGB324 is a selective inhibitor of the Axl receptor tyrosine kinase, which blocks epithelial-mesenchymal transition, a key driver in drug-resistance and metastasis.


The Orphan Drug designation-to encourage development of drugs in the diagnosis, prevention, or treatment of a medical condition affecting fewer than 200,000 people in the U.S.-grants a product market exclusivity for a seven-year period if the sponsor complies with certain FDA specifications, as well as tax credits and prescription drug user fee waivers. The designation does not, though, shorten the duration of the regulatory review and approval process.


Approximately 18,860 new cases of AML were diagnosed in the U.S. in 2014, and approximately 10,460 individuals died as a result of the disease, according to American Cancer Society estimates.


BGB324 is currently being evaluated in a multicenter Phase Ib trial for patients with AML as both a single agent and in combination with cytarabine (the current standard of care drug for patients with AML).


Endpoints for the trial include safety, tolerability, clinical response, and assessment of novel biomarkers. Data are expected from the trial in 2015, and additional trials with the drug in non-small cell, lung cancer are in preparation, according to a news release from the drug's manufacturer, BerGenBio AS.