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FDA grants orphan drug designation to tazemetostat for soft tissue sarcoma
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The FDA granted orphan drug designation to tazemetostat for the treatment of patients with soft tissue sarcoma, according to the drug’s manufacturer.
Tazemetostat (Epizyme), a first-in-class EZH2 inhibitor, is being evaluated in an ongoing phase 2 study for the treatment of follicular lymphoma, diffuse large B-cell lymphoma (DLBCL), mesothelioma and certain molecularly defined solid tumors.
“This is an important milestone for Epizyme as we advance tazemetostat through clinical development,” Robert Bazemore, president and CEO of Epizyme, said in a company-issued press release.
The FDA previously granted fast track designation to tazemetostat for patients with follicular lymphoma regardless of EZH2 mutation status, and for patients with DLBCL who harbor EZH2–activating mutations. The FDA also granted orphan drug designation to tazemetostat for the treatment of soft tissue sarcoma and malignant rhabdoid tumors.
“We are encouraged by the positive regulatory milestones we have achieved for tazemetostat, including this orphan drug designation for soft tissue sarcomas,” Bazemore said. “We look forward to our continued engagement with the FDA as we work to bring tazemetostat to patients with both solid tumors and hematological malignancies as quickly as possible.”
The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.