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FDA Grants Orphan Drug Designation to Mocetinostat for Diffuse Large B-Cell Lymphoma

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The U.S. Food and Drug Administration has granted Orphan Drug Designation to Mirati Therapeutics’ mocetinostat, a spectrum-selective HDAC inhibitor, for diffuse large B-cell lymphoma. In June, mocetinostat was granted Orphan Drug Designation as a treatment for myelodysplastic syndrome.  Orphan drug designation is also being sought for bladder cancer patients with specific genetic alterations.

Mocetinostat is being developed as a single-agent treatment in patients with diffuse large B-cell lymphoma and bladder cancer with specific genetic mutations in histone acetyl transferases (HATs) that are believed to be critically involved in the pathogenesis and progression of these tumor types.  The agent reverses aberrant acetylation resulting from HAT mutations and is predicted to halt tumor progression and reduce tumor burden in patients.

"We have identified genetic alterations in histone acetylation pathways (CREBBP and EP300) in approximately one-third of [diffuse large B-cell lymphoma] and bladder tumors. Nonclinical tumor models exhibiting these mutations are particularly responsive to mocetinostat," said Charles Baum, MD, PhD, President and CEO of Mirati.

Mirati is currently enrolling patients in phase II clinical studies of mocetinostat in combination with azacitadine for intermediate-risk and high-risk myelodysplastic syndrome. Phase II studies of single-agent mocetinostat are being planned in patients with HAT mutations in bladder cancer and diffuse large B-cell lymphoma.

The content in this post has not been reviewed by the American Society of Clinical Oncology, Inc. (ASCO®) and does not necessarily reflect the ideas and opinions of ASCO®.


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