The Food and Drug Administration will review an investigational drug for a rare type of cancer that affects soft tissues under the skin.
Cambridge, Massachusetts-based Epizyme said Thursday that the agency had accepted its application for accelerated approval of the drug tazemetostat in metastatic or locally advanced epithelioid sarcoma ineligible for curative surgery, based mainly upon data from 62 patients in a Phase II study. The Prescription Drug User Fee Act target date – the FDA’s deadline to reach a decision on whether or not to approve the drug – is Jan. 23, 2020.
Epizyme’s shares fell nearly 11 percent on the Nasdaq when markets opened Thursday, but quickly recovered their value and are currently up nearly 1 percent from their Wednesday closing price.
Data from the 62 patients were shown in an oral presentation at the American Society of Clinical Oncology’s annual meeting last month. The data showed a 15 percent objective response rate and 26 percent disease control rate, while the median duration of response had not yet been reached. The drug was described as well-tolerated with favorable safety. The drug is an EZH2 inhibitor being investigated as a treatment for multiple solid tumors, as well as follicular lymphoma.
“This is a significant achievement in the development of this potentially first-in-class EZH2 inhibitor, and we look forward to working with FDA during the review,” Epizyme CEO Robert Bazemore said in a statement. “If approved, we believe tazemetostat could become an important new option in the treating physician’s arsenal.”
In a note to investors Thursday, Cowen analyst Yaron Werber wrote that the drug is approvable given high unmet need and a favorable risk-reward profile from the data. AT the same time, he wrote, it is likely that the FDA will call a meeting by the Oncologic Drugs Advisory Committee, or ODAC, which would probably occur in the fourth quarter of this year. The FDA convenes AdCom meetings when it requires additional expertise from outside experts as it reviews a drug. It is not required to follow their recommendations whether or not to approve a drug, but usually does.
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
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