Mocetinostat Receives Orphan Drug Status

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The U.S. Food and Drug Administration (FDA) has granted orphan drug status to mocetinostat, a spectrum selective histone deacetylase (HDAC) inhibitor for the treatment of diffuse large B-cell lymphoma (DLBCL). Mocetinostat was also granted orphan drug status for the treatment of myelodysplatic syndrome (MDS) in June, and Mirati Therapeutics, Inc., developer of mocetinostat, is also seeking orphan drug status for bladder cancer in patients with certain genetic mutations.


Mirati Therapeutics is developing mocetinostat as a single agent for the treatment of DLBCL and bladder cancer with genetic alterations involving histone acetyl transferases (HATs) that are thought to have a significant role in the pathogenesis of these cancer types. Mocetinostat reverses abnormal acetylation caused by these HAT mutations, thereby halting cancer progression and reducing tumor burden. Researchers have identified HAT mutations in about one-third of patients with DLBCL and bladder cancer.


Mocetinostat is also being studied in a phase 2 trial in combination with azacitidine for the treatment of intermediate and high-risk MDS. Initial data from this phase 2 study are expected by the end of 2014. In addition, researchers have completed 13 clinical trials involving mocetinostat in more than 400 patients with a variety of solid and hematologic malignancies.

Preventive therapy for ER-negative breast cancer
Preventive therapy for ER-negative breast cancer

Mirati Therapeutics, Inc. today announced that the U.S. FDA has granted Orphan Drug Designation to mocetinostat, a spectrum selective HDAC inhibitor, for diffuse large B-cell lymphoma (DLBCL)

In June, mocetinostat was granted Orphan Drug Designation as a treatment for myelodysplastic syndrome (MDS). Orphan drug designation is also being sought for bladder cancer patients with specific genetic alterations.


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