DLBCL Drug from Mirati Earns Orphan Drug Designation

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The US Food and Drug Administration has granted Orphan Drug Designation to a treatment under development for diffuse large B-cell lymphoma.

Mirati Therapeutics Inc. made the announcement yesterday regarding mocetinostat, a spectrum selective HDAC inhibitor. Earlier this summer, the compound was granted Orphan Drug Designation as a treatment for myelodysplastic syndrome (MDS). The company seeks the same designation for certain bladder cancer patients.

Mocetinostat is being developed as a single agent treatment in patients with diffuse large B-cell lymphoma (DLBCL) and bladder cancer with specific genetic mutations in Histone Acetyl Transferases (HATs) the company believes to be critically involved in the development and progression of these cancers. It is believed that the compound reverses some of the processes involved after a cell mutates and becomes cancerous and the company believes it can halt tumor progression and reduce tumor burden in patients.

Mocetinostat is currently also in Phase II clinical studies in combination with Vidaza as a treatment for intermediate and high-risk MDS.

"We have identified genetic alterations in histone acetylation pathways (CREBBP and EP300) in approximately one third of DLBCL and bladder tumors. Nonclinical tumor models exhibiting these mutations are particularly responsive to mocetinostat," said Dr. Charles Baum, president and CEO of Mirati. "Among other benefits, orphan designation provides seven years of market exclusivity to target these genetically defined patients with unmet medical need in the event we achieve regulatory approval."

Source: Mirati Therapeutics

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