FDA Removes Partial Clinical Hold on Tazemetostat Clinical Trials

Epizyme is reopening enrollment in trials in the US.

Cambridge, MA-based biotech company Epizyme, Inc. is developing epigenetic medicines that operate via new mechanisms of action. Since 2011, the company has discovered five novel epigenetic therapies, three of which are in clinical development and two that have begun or will soon begin IND-enabling studies.

Its lead candidate tazemetostat is a first-in-class, potent, selective, orally available EZH2 inhibitor with studies underway as a monotherapy in ongoing Phase 2 programs in certain molecularly defined solid tumors, including; follicular lymphoma (FL); and combination studies in diffuse large B-cell lymphoma (DLBCL) and non–small cell lung cancer (NSCLC).

In April 2018, the US Food and Drug Administration (FDA) placed a clinical hold on recruitment into the U.S. trial of Epizyme, Inc.'s tazemetostat following reports of a child with advanced poorly differentiated chordoma, a rare bone cancer, developing a secondary T-cell lymphoma.

The agency removed the partial hold in late September after Epizyme conducted a comprehensive assessment of the risk of secondary malignancies associated with tazemetostat, taking into consideration published literature and the company’s clinical experience to date. A panel of external scientific and medical experts also reviewed and validated the findings regarding the efficacy and safety data of all of Epizyme’s trials in hematological malignancies and solid tumors in adults and children. 

Epizyme is in the process of reopening enrollment in its trials in the U.S., including the follicular lymphoma (FL) EZH2 activating mutation cohort of its Phase 2 non-Hodgkin lymphoma trial.

“The Epizyme team has worked diligently to provide a comprehensive response back to the FDA, and through constructive dialogue, we successfully resolved the partial clinical hold. This allows us to turn our full attention to our key priorities: preparing for our first NDA submission for tazemetostat in epithelioid sarcoma and defining our registration path in FL,” said Robert Bazemore, President and Chief Executive Officer of Epizyme.

The company is also working with regulators in France and Germany to get the partial clinical holds on trials in those countries removed.

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