FDA grants orphan drug status for BerGenBio’s BGB324 to treat acute myeloid leukaemia

Recently, the company has also started a multi-centre Phase Ib trial of BGB324, a selective inhibitor of Axl, in AML patients.

The two-part trial is primarily designed to evaluate the safety and tolerability of BGB324 when administered as a single agent and in combination with a standard-of-care drug (cytarabine).

The secondary endpoints of the trial will explore evidence of clinical response and evaluate new biomarkers.

The trial will be conducted at six sites in Norway, Germany and the US, while the results are expected to be available in 2015.

BerGenBio chief executive officer Richard Godfrey said: "The FDA’s decision to grant BerGenBio orphan-drug status for BGB324 is a significant milestone for the company and recognises the need for innovative new ways to treat AML.

"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 US."

According to the company, additional clinical trials with BGB324 in non-small cell lung cancer (NCCLC) are in preparation.

BGB324 is a first-in-class, highly selective small molecule inhibitor of the Axl receptor tyrosine kinase that blocks the epithelial-mesenchymal transition (EMT), a key driver in drug-resistance and metastasis.