Tox issues? Check. Questionable efficacy data? Check. OK to market? ODAC says check that box for Epizyme
Based on the FDA’s own in-house review, Epizyme doesn’t have a whole lot of evidence to prove tazemetostat works well in fighting epithelioid sarcoma, which definitely runs hot in triggering serious side effects and has that FDA hold in its clinical past to raise additional concerns.
But everyone on the Oncologic Drugs Advisory Committee thought it was fine for marketing. They voted 11 to 0 to urge the FDA to give Epizyme $EPZM the green light.
Yaron Werber at Cowen noted:
Overall, the committee concluded that disease stabilization, durability of response, and clean safety/tolerability of taze were enough to merit approval. While the panel was also critical about the confirmatory Ph3 trial design, in our conversations with Epizyme, they noted that FDA agreed that enrollment of the study will take a long time and that PFS is a valid endpoint (ie OS will be too hard to assess).
The vote may not surprise even the most skeptical analysts, like Michael Yee at Jefferies, who only gave it a 20% to 25% chance of success at the agency. Without much to choose from now, physicians like as many options as they can get on cancer. And for very sick patients, risks can be a secondary issue, even if 34% of tazemetostat patients had to suspend dosing due to toxicity.
That flexible perspective is typically on view at ODAC, which has a built-in bias in favor of approvals that can sustain even the most marginal drugs.
The vote clearly makes it hard for the FDA to say no now, though they are free to do as they choose. And the favorable sentiment for the drug on ODAC also bolsters Epizyme’s upcoming shot at an OK for follicular lymphoma, where the biotech stands to make a potential $500 million-plus in peak sales.
Investors certainly like the sound of everything. They drove Epizyme shares up 10% on Wednesday.