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Epizyme wins FDA approval for epithelioid sarcoma treatment

Phase 2 clinical trial shows response rates outweigh risks

FDA

The US Food and Drug Administration (FDA) has approved Epizyme’s Tazverik (tazemetostat) for the treatment of epithelioid sarcoma.

Epizyme won accelerated approval for the use of the small molecule inhibitor of the histone-lysine methyltransferase EZH2 gene in patients aged 16 years and older who have metastatic or locally advanced forms of the rare soft tissue disease and are ineligible for a complete resection.

The FDA approved Tazverik on the strength of data from an open-label, single-arm cohort of a phase 2 clinical trial. That cohort enrolled patients with epithelioid sarcoma, while other arms of the study targeted additional indications including synovial sarcoma and renal medullary carcinoma.

Epizyme enrolled 62 patients in the cohort and gave them 800 mg of Tazverik twice a day until their disease progressed or they suffered unacceptable toxicity. One patient experienced a complete response. A further eight patients had partial responses, resulting in an objective response rate of 15%.

In another group of epithelioid sarcoma patients with similar characteristics, which Epizyme enrolled to assess the pharmacodynamics of Tazverik, the rates of complete and partial responses were 2% and 9%, respectively.

Ahead of an advisory committee session focused on Tazverik, the FDA asked the experts it gathered to consider whether those response rates, coupled to duration of responses running from three to 24 months, were compelling enough to outweigh the risks posed by the drug. Almost half of patients in the dataset analysed by the FDA experienced a Grade 3 or 4 adverse reaction.

The advisory committee was unanimous in its decision, voting by 11-0 in favour of approving the drug. While only a small minority of patients responded to Tazverik, the panellists felt the evidence that some patients with very limited treatment options had durable responses was strong enough to justify approving the drug.

Other sarcoma drugs, such as Novartis’ Votrient and Eli Lilly’s Lartruvo, are not specifically indicated for use in epithelioid sarcoma.

This week the FDA concurred with its advisory committee and approved Tazverik, while placing some responsibilities on Epizyme in light of the limitations of the data currently available.

Epizyme needs to verify the clinical benefit of Tazverik by running a confirmatory trial. That study, a randomised controlled trial that is assessing the benefits of adding Tazverik to doxorubicin, is already underway.

The FDA has also asked Epizyme to assess the effect of Tazverik on liver function as part of a package of post-marketing requirements. Epizyme also plans to expand enrollment in the phase 2 cohort it set up to assess pharmacodynamics with a view to gathering data to support a label change.

Article by
Nick Taylor

24th January 2020

From: Research

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