FDA waves Epizyme's $186K rare cancer drug through to market — now get ready for the second act
After winning the hearts of the expert panel convened by the FDA despite a bleak in-house review and a checkered development history, Robert Bazemore has steered Epizyme to its first-ever OK for a rare cancer drug.
The approval in epithelioid sarcoma sets tazemetostat, now Tazverik, up nicely for a quick expansion to follicular lymphoma — a much bigger indication for which the biotech has just submitted an NDA.
With that and other ambitions in mind, Epizyme has priced the drug — an inhibitor of the EZH2 enzyme — at $15,500 per month, or $186,000 for a year. A field-based team of 19 is already in place to push Tazverik to a specialty distribution network within 10 days, chief strategy & business officer Matthew Ros said in a conference call.
Epithelioid sarcoma, or ES, is an extremely rare subtype of soft tissue sarcoma. Per Epizyme estimates around 800 are living with the condition in the US, many of them young adults. Tazverik’s label covers its use in patients 16 or older with metastatic or locally advanced disease who are not eligible for the standard treatment: complete resection.
“There are 300 ES, INI- patients in the U.S. who qualify for the label,” Cowen analyst Yaron Werber wrote in a note. “Our estimates predict market share rising from 8% in FY20 to 65% in FY25.”
Epizyme’s success here once again underscores the FDA’s willingness to hand out accelerated approvals in the face of tough cancers that leaves patients with few to no options. In the single-arm study that formed the basis of approval, the overall response rate was 15% among 62 patients who took 800 mg of Tazverik twice a day. That’s a total of nine patients. Meanwhile, 37% had a serious adverse event and 34% needed to suspend dosing due to toxicity. And that’s after Epizyme had to contend with a clinical hold and a decision to scrap the monotherapy program in diffuse large B-cell lymphoma.
But the FDA highlighted how 50% of ES patients would have had metastatic disease at the time of diagnosis, which would be life-threatening.
“Epithelioid sarcoma accounts for less than one percent of all soft tissue sarcomas,” Richard Pazdur, the powerful director of the FDA’s Oncology Center of Excellence, remarked. “Until today, there were no treatment options specifically for patients with epithelioid sarcoma.”
Follicular lymphoma (FL) is a much different story, with multiple players equipped with new tech such as CAR-T and bispecifics vying for the market. But Epizyme believes it has gotten a head start.
“This is a very efficient NDA,” CMO Shefali Agarwal said regarding their submission for third-line follicular lymphoma. “It’s a small NDA because we cross referenced most of the other modules like preclinical, CMC and clinical pharmacology, so all that has been reviewed with ES. Now all that’s left in the NDA is just the clinical aspect.”
Michael Yee of Jefferies has predicted that Tazverik can bring in $500 million-plus in peak sales as an FL therapy — while ES is not really in the model.