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BerGenBio Drug Gets Orphan-Drug Status

BerGenBio AS, a Norwegian oncology biopharmaceutical company, announced Wednesday that the US Food and Drug Administration (FDA) granted orphan-drug designation for BGB324 for treatment of acute myeloid leukemia (AML).

Earlier this month BerGenBio announced that the first patient was dosed in its multi-center Phase 1b trial of BGB324, a selective inhibitor of Axl, 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 study will be conducted at six sites in Norway, Germany and the United States.

 BerGenBio expects data to be available from this trial in 2015.


 “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,” said Richard Godfrey, chief executive officer of BerGenBio. “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 seven years of market exclusivity in the United States.”