Dive Brief:
- The Food and Drug Administration has cleared a new kind of cell therapy, granting late Friday an accelerated approval to Iovance Biotherapeutics’ Amtagvi for people with an advanced form of skin cancer.
- Amtagvi is the first marketed cellular medicine made by engineering immune cells known as tumor-infiltrating lymphocytes, or TILs. It’s for people whose melanoma can’t be surgically removed or has spread to other parts of the body, and is meant to be used after other medicines.
- The FDA’s OK was based on data showing Amtagvi shrank tumors in testing, a benefit that must be confirmed in an ongoing Phase 3 trial. Iovance set the drug’s list price at about $515,000, the highest of any cell-based medicine for cancer in the U.S. Its prescribing information includes warnings for the risk of low blood cell counts, severe infections or kidney problems.
Dive Insight:
Amtagvi’s approval has been a long time coming for Iovance.
TIL therapy originated from research at the National Institutes of Health in the 1980s. It took decades to fine-tune, and Iovance had to overcome multiple regulatory delays bringing the therapy, formerly known as lifileucel, to market.
Its clearance is the “culmination of scientific and clinical research efforts,” said Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, in a statement.
An estimated 8,000 people die from melanoma each year, according to Iovance. Until now, there haven’t been drugs available for people whose cancers progress after a commonly used immunotherapy or targeted cancer medication.
Iovance must now prove it can manufacture and deliver a cell therapy, a complicated task that has challenged even large drugmakers like Gilead Sciences, Novartis and Bristol Myers Squibb, which sell a different kind of cell therapy for a range of blood cancers.
Amtagvi is derived from a tumor sample that’s surgically removed. Select immune cells that have “infiltrated” the tumor are isolated, grown and filtered via a weekslong manufacturing process. It currently takes Iovance 34 days from the time a sample is received to make and ship back each personalized TIL therapy — longer than with the CAR-T cell therapies from Gilead, Novartis and Bristol Myers.
“We think we can improve on it over time,” said CEO Frederick Vogt, on a conference call with analysts.
Executives said 30 treatment centers will be active when the launch begins, climbing to over 50 within 90 days of the FDA’s original approval decision date of Feb. 24.
Still, Stifel analyst Benjamin Burnett noted there’s some questions among experts as to how many of those centers are currently capable of administering Amtagvi. There’s also debate among investors about demand and the complex logistics involved.
“Cell therapy launches have been clunky,” Burnett wrote in a research note.
For its part, the company anticipates “strong market access and patient reimbursement” given early response from payers, Jim Ziegler, the company’s executive vice president of commercial, said. The majority of patients are on commercial insurance, he said.
“We expect a bolus of patients coming through as the product launches and we start to see the manufacturing flow,” said Vogt.
Iovance wouldn’t detail how much revenue it expects to earn. But the company’s current manufacturing capacity and pricing imply Amtagvi could generate $185 million in its first three quarters on the market, wrote Jefferies analyst Michael Yee.
“Consensus numbers look doable,” Yee wrote in a client note, adding that Iovance “would make a nice tuck-in for big pharma, who could leverage this even better.”
Iovance shares climbed about 35% in early trading Tuesday.