FDA Lifts Clinical Hold on Epizyme’s Lead Candidate’s Study

FDA Lifts Clinical Hold on Epizyme’s Lead Candidate’s Study

The FDA has lifted a partial clinical hold on Epizyme’s cancer candidate tazemetostat. A small molecule EZH2 inhibitor, tazemetostat, holds the promise for treating aggressive cancers such as leukemia, lymphoma, and multiple myeloma. Back in April, the FDA paused enrollment of new patients in clinical trials after one child in the study developed lymphoblastic leukemia.


The FDA lifted the hold imposed in April only after Epizyme provided comprehensive evidence that tazemetostat won’t lead to secondary lymphomas. The company also turned to an independent group of scientific and medical experts to validate the cancer drug’s efficacy and safety.

Epizyme, a Massachusetts-based biotech company, is now in the process of reopening enrollment in all of its company-sponsored studies in the United States. Their aim is for tazemetostat to be prescribed as a treatment for epithelioid sarcomas.

Sarcoma is a rare and difficult to cure cancerous tumor of the connective tissues. Over 5,000 Americans are expected to die of soft tissue sarcomas in 2018 and other 13,000 new patients will be diagnosed with this cancer, according to recent estimates from The American Cancer Society.

The FDA’s announcement was immediately reflected in stock prices. Shares in Epizyme rose 25% in premarket trade. The hold lift on tazemetostat is part of the FDA’s bigger effort to accelerate approvals for breakthrough drugs.

The agency has also recently approved therapies that once seemed futuristic, including  Biotronik’s PK Papyrus stent, meant to treat acute coronary artery perforation, a rare heart complication, is the first FDA approved device of its kind in 17 years.

The FDA’s commitment to keep up with technological innovation was best showcased in July, at AcademyHealth’s 2018 Health Datapalooza. Commissioner Scott Gottlieb announced more efficient regulatory processes for companies working on artificial intelligence powered devices that will propel the digital transformation of healthcare.

Read the latest interview from Simon Stertzer about biotechnology’s impact on treatment and prevention.

Powered by