The Food and drug Administration has approved the first drug for a rare form of connective-tissue cancer.
Cambridge, Massachusetts-based Epizyme said Thursday that the FDA had given accelerated approval Tazverik (tazemetostat) for epithelioid sarcoma, or ES, in patients whose disease is ineligible for complete surgical removal. The FDA’s Oncologic Drugs Advisory Committee had voted unanimously to recommend approval for the drug in its meeting last month.
“Our commercial launch plans are underway, and we expect to make Tazverik available to ES patients and treating physicians across the U.S. within 10 business days,” Epizyme CEO Robert Bazemore said in a statement.
Tazverik is an EZH2 inhibitor that is also in development for follicular lymphoma. Shares of the company were down more than 12% in late-morning trading on the Nasdaq Friday.
The approval was based on data from a Phase II study showing a 15% overall response rate and median 70 weeks’ duration of response, along with a low percentage of adverse events. Sarcomas are a broad spectrum of cancers affecting the connective tissues of the body. ES makes up less than 1% of a subgroup known as soft-tissue sarcomas, which are already rare. The company is also running a Phase III confirmatory trial assessing the drug in frontline ES, comparing the combination of Tazverik and the chemotherapy doxorubicin against doxorubicin and placebo.
Despite the slump in the company’s stock price following the news, Cowen analyst Yaron Werber was upbeat in a note to investors. The label, he noted, is clean, without a black-box warning and thus offering reassurance about any potential risk of secondary malignancies arising from use of the drug. While the initial launch will be modest, he wrote that it is likely to accelerate assuming an approval for follicular lymphoma. The drug’s list price of $186,000 per year is also in line with estimates.
The company submitted its application for FDA approval in follicular lymphoma last month. The application, which would also be for accelerated approval, was based on data presented at the American Society of Hematology’s annual meeting earlier in the month, showing a 69% overall response rate among 45 patients who had EZH2 mutations and 35% among 54 who did not. Median durations of response in the two groups were 11 and 13 months, respectively, while median progression-free survival – the amount of time a patient goes without dying or their disease worsening – was 14 and 11 months. The company is also launching a Phase III confirmatory study to support the drug’s full approval.
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