- Gilead's cancer cell therapy Tecartus is now approved in the U.S. to treat adults with acute lymphoblastic leukemia, the treatment's second clearance by the Food and Drug Administration and the first for a so-called CAR-T drug in people with the blood cancer who are older than 25.
- The approval, announced by Gilead's Kite Pharma unit on Friday, follows a little more than a year after Tecartus first secured FDA clearance for a type of lymphoma. Tecartus is Kite's second CAR-T therapy after Yescarta, which was launched in 2017.
- Novartis also markets its CAR-T therapy Kymriah for acute lymphoblastic leukemia, or ALL, but only for children and young adults up to age 25. Tecartus' approval overlaps partially with Kymriah's, as it's cleared for adults older than 18 years.
Tecartus' new indication is Gilead's fourth in cell therapy and its first in leukemia following previous approvals for Yescarta in diffuse large B-cell and follicular lymphoma and for Tecartus in mantle cell lymphoma.
The approvals mark a steady expansion for Gilead's Kite unit, which the larger biotech bought in 2017 for nearly $12 billion. Yet while sales of Yescarta and now Tecartus have grown, the cell therapy business has struggled to live up to expectations set when Gilead acquired Kite. Gilead has twice written down the value of the research assets obtained the buyout.
Still, the larger market opportunity unlocked by the additional approvals has helped boost uptake, and total CAR-T sales are pacing at nearly $900 million per year. A good part of that financial performance is due to the therapies' extremely high price: both Yescarta and Tecartus cost $373,000 per patient. (The list price of Tecartus is the same in ALL as it is in MCL, a company spokesperson confirmed.)
Treatment, which involves a complex procedure to genetically reengineer patients' own immune cells, is meant to deliver a long-lasting benefit. For some, that's the case, although in others treatment doesn't work as well or leads to severe side effects that are often associated with cell therapy.
The approval of Tecartus in adult ALL was based on data from a single-arm Phase 1/2 study. Overall, about 65% experienced complete remission after a follow-up of roughly one year. Remission lasted a median of just over 13 months, although for some patients it extended closer to two years.
A quarter of 78 patients treated at the target dose of Tecartus experienced Grade 3 or higher cytokine release syndrome, a dangerous overactivation of the immune system, and 35% had Grade 3 or higher neurologic side effects.
"Roughly half of all ALL cases actually occur in adults, and unlike pediatric ALL, adult ALL has historically had a poor prognosis," said Lee Greenberger, chief scientific officer of The Leukemia & Lymphona Society, in a statement provided by Gilead.
In the study Novartis used to win approval of Kymriah in pediatric ALL, 83% went into complete remission following treatment.
Eligible patients can receive Tecartus at over 100 treatment centers across the U.S., although many are clustered on the East and West coasts as well as around the Great Lakes.
For treatment, patient cells are extracted from the blood, frozen and shipped to a Gilead manufacturing site, where they are modified to seek out a certain protein commonly expressed on leukemia and lymphoma cells. After they're reengineered and tested, the cells are frozen and shipped back to the treating site, where physicians reinfuse them back into the patient.
In the study supporting Tecartus' approval, Gilead was able to return a completed CAR-T product to patients in 16 days, measured from when cells were extracted to when they were reinfused, the spokesperson said in an email.
Note: This story has been updated with additional detail on Tecartus' pricing and manufacturing.