US-based pharmaceutical company Mirati Therapeutics has received orphan drug designation from the US Food and Drug Administration (FDA) for its mocetinostat for diffuse large B-cell lymphoma (DLBCL).
Mocetinostat is an orally bioavailable and spectrum-selective HDAC inhibitor that is being developed as a single agent treatment in patients with DLBCL and bladder cancer with specific genetic mutations in histone acetyl transferases (HATs).
The drug candidate is believed to reverse aberrant acetylation resulting from HAT mutations and prohibit tumour progression and reduce tumour burden in patients.
Mirati Therapeutics president and CEO Charles Baum said: “We have identified genetic alterations in histone acetylation pathways (CREBBP and EP300) in approximately one third of DLBCL and bladder tumours. Nonclinical tumour models exhibiting these mutations are particularly responsive to mocetinostat.
“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.”
Mocetinostat is currently in Phase II clinical studies in combination with Vidaza as a treatment for intermediate and high-risk MDS.
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The company is also enrolling patients to initiate Phase II studies of mocetinostat as a single agent in patients with mutations in histone acetyl transferases in bladder cancer and DLBCL.
Initial results from the Phase II studies are expected to be presented by the end of the year.
Mocetinostat has completed 13 clinical trials to date in more than 400 patients with different hematologic malignancies and solid tumours.
In June, mocetinostat received orphan drug designation as a treatment for myelodysplastic syndrome (MDS).
The company is also trying to receive orphan drug designation for mocetinostat to treat bladder cancer patients with specific genetic alterations.
Image: Micrograph of a diffuse large B-cell lymphoma. Photo: courtesy of Nephron.