MGCD0103 is an orally-administered, isotype-selective HDAC inhibitor. The compound is currently in one Phase I combination clinical trial with Taxotere for solid tumors, two Phase I/II combination trials with Vidaza for hematological malignancies and with Gemzar for pancreatic cancer, and five Phase II clinical trials in hematological malignancies.
Orphan drug designation allows special incentives for sponsors planning to test a product for use in a rare disease or condition. Once approved, the product may qualify for seven years of marketing exclusivity in the US.