Epizyme's leukemia compound granted orphan drug designation

19 August 2013

US biotech company Epizyme (NASDAQ: EPZM) has been granted orphan drug designation for its experimental compound EPZ-5676 by the US Food and Drug Administration.

The company is developing EPZ-5676, a small molecule inhibitor of DOT1L created with Epizyme’s proprietary product platform, for the treatment of acute leukemias in which the MLL gene is rearranged due to a chromosomal translocation (MLL-r).

Epizyme retains all US rights to EPZ-5676 and has granted US biotech firm Celgene (Nasdaq: CELG) an exclusive license to EPZ-5676 outside of the USA. Epizyme has also partnered with USA-based Abbott Laboratories (NYSE: ABT) to develop a companion diagnostic to identify MLL-r patients.

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