Mirati Therapeutics' Mocetinostat Granted FDA Orphan Drug Designation For MDS

(RTTNews) - Mirati Therapeutics, Inc. (MRTX) said Tuesday that mocetinostat, the company's spectrum selective HDAC inhibitor, has been granted Orphan Drug Designation by the U.S. Food & Drug Administration as a treatment for myelodysplastic syndrome.

Mocetinostat is being developed in Phase 2 clinical studies in combination with Vidaza as a treatment for intermediate and high-risk myelodysplastic syndrome, as well as a single agent treatment in patients with diffuse large B-cell lymphoma and bladder cancer targeting specific genetic mutations in histone acetylation that increase the likelihood of response in tumor cells.

The FDA's Office of Orphan Drug Products grants orphan status to support development of medicines for underserved patient populations or rare disorders that affect fewer than 200,000 people in the United States. Orphan drug designation provides certain benefits, including market exclusivity upon regulatory approval if received, exemption of FDA application fees and tax credits for qualified clinical trials.