Tazemetostat Granted Orphan Drug Designation for the Treatment of Soft Tissue Sarcoma

Epizyme announced the U.S. Food and Drug Administration (FDA) granted Orphan Drug designation to tazemetostat, the company’s first-in-class EZH2 inhibitor, for the treatment of patients with soft tissue sarcoma (STS).

"This is an important milestone for Epizyme, as we advance tazemetostat through clinical development," said Robert Bazemore, president and chief executive officer, Epizyme. "We are encouraged by the positive regulatory milestones we have achieved for tazemetostat, including this Orphan Drug designation for soft tissue sarcomas. 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 Orphan drug status conveys eligibility for certain development incentives and market exclusivity for STS independent from Epizyme’s intellectual property protection. The FDA Orphan Drug Designation program provides orphan status to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the U.S.

STS is a cancer occurring in muscle, fat, blood vessels, tendons, fibrous tissues and connective tissue, which can arise anywhere in the body at any age. STS is an aggressive and difficult-to-treat cancer with more than 50 subtypes, including epithelioid sarcoma, highlighting the need for effective treatment options. According to the American Cancer Society, approximately 13,000 patients will be diagnosed with STS in the U.S. in 2017.

Tazemetostat, a first-in-class EZH2 inhibitor, is currently being studied in ongoing Phase 2 programs in both follicular lymphoma and diffuse large B-cell lymphoma (DLBCL) forms of non-Hodgkin lymphoma; certain molecularly defined solid tumors, including epithelioid sarcoma and other INI1-negative tumors; and mesothelioma, as well as in combination studies in DLBCL. Tazemetostat has been granted Fast Track designation by the U.S. Food and Drug Administration for follicular lymphoma regardless of EZH2 mutation and for DLBCL with EZH2-activating mutations, as well as Orphan Drug designation for soft tissue sarcoma and malignant rhabdoid tumors.