FDA Grants Orphan Drug Status to Investigational Acute Myeloid Leukemia Treatment

BerGenBio AS announced that the FDA has granted Orphan Drug designation to BGB324 for the treatment of acute myeloid leukemia (AML).

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Earlier this month BerGenBio announced that the first patient has been dosed in its multi-center Phase 1b 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). The company plans to announce data from this trial in 2015. Additional clinical trials with BGB324 in non-small cell lung cancer are currently underway.

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.

For more information visit BerGenBio.com.