A drug for treating a rare form of connective-tissue cancer looks likely to win Food and Drug Administration approval after a unanimous positive vote by a panel of experts.
The FDA’s Oncologic Drugs Advisory Committee voted 11-0 to recommend accelerated approval for Cambridge, Massachusetts-based Epizyme’s tazemetostat for epithelioid sarcoma, a form of soft-tissue sarcoma.
Shares of Epizyme rose 35.6% on the Nasdaq Wednesday following the news, before leveling off and by Thursday afternoon trading at around 8% above their Wednesday closing price.
“We are incredibly pleased by ODAC’s unanimous support for the benefit-risk of tazemetostat in ES, and we appreciate the tremendous support received from sarcoma physicians and their medical teams, advocates, caregivers and – most notably – patients with ES,” Epizyme Chief Medical Officer Shefali Agarwal said in a statement. “We believe in the strength of the totality of data – including the observed durable responses and stabilization of disease, safety and tolerability – are distinguishing characteristics of tazemetostat for patients living with this disease.”
The drug is an EZH2 inhibitor that is also being explored in other cancers. Also on Wednesday, Epizyme applied for FDA approval of the drug in follicular lymphoma.
The submission for ES was based primarily on cohort data from 62 patients in the Phase II trial Study 202. The overall response rate was 15%, and 63% of patients had a complete response, partial response or stable disease as their best response. Median duration of response was 70 weeks. Overall, according to the ODAC briefing document, there was a low percentage of adverse side effects leading to discontinuation or dose reduction, and side effects overall were manageable.
Soft-tissue sarcomas are already rare, and ES accounts for less than 1% of them, occurring mostly in young men and affecting the extremities. The briefing document noted that there are two drugs – namely the chemotherapy agent doxorubicin and Novartis’ Votrient (pazopanib) – that have broad labels for soft-tissue sarcomas and won approval based on clinical trials with 10 or fewer ES patients.
In a note to investors, Cowen analyst Yaron Werber wrote the company is finalizing commercial plans for a launch of the drug in the first quarter of 2020, with PI3K inhibitors and Bristol-Myers Squibb’s Revlimid (lenalidomide) being considered as benchmarks for tazemetostat’s price. According to Revlimid manufacturer Celgene, which BMS acquired earlier this year, the wholesale acquisition cost for a 28-capsule bottle of Revlimid is $20,154.94.
Photo: FDA
