FDA ushers in a new era in cancer treatment with ‘historic’ CAR-T approval for Novartis

The FDA has approved the world’s first CAR-T therapy, giving a green light to Novartis for Kymriah (tisagenlecleucel) in what regulators themselves describe as an historic event.

The early approval — about a month ahead of the PDUFA date — came through for certain pediatric and young adult patients with a form of acute lymphoblastic leukemia.

Novartis set the price for the one-time treatment at $475,000, right in line with low-end estimates and one that will put pressure on Gilead to rein in its own price for a rival therapy expected to be approved in the near future.

Scott Gottlieb, FDA Commissioner

None other than FDA commissioner Scott Gottlieb did the honors in the agency’s statement, noting:

We’re entering a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack a deadly cancer. New technologies such as gene and cell therapies hold out the potential to transform medicine and create an inflection point in our ability to treat and even cure many intractable illnesses. At the FDA, we’re committed to helping expedite the development and review of groundbreaking treatments that have the potential to be life-saving.

A group of biopharma companies have been racing to this breakthrough point. Kite Pharma $KITE, newly bought out in a $12 billion deal by Gilead $GILD, is coming in a close second with its own application for axicel.

Novartis’ investigators registered a game-changing 83% remission rate in its pivotal study.

CAR-T, though, is also associated with severe and potentially deadly side effects, including lethal instances of cytokine release syndrome with some patients dying from brain swelling in separate studies from the Novartis drug.

Regulators noted that they will require special training for anyone involved in delivering this therapy, while expanding the approval of Actemra (tocilizumab) to treat CAR T-cell-induced severe or life-threatening CRS in patients 2 years of age or older. “In clinical trials in patients treated with CAR-T cells,” the FDA reported, “69% of patients had complete resolution of CRS within two weeks following one or two doses of Actemra.”

This therapy, which is made using a patient’s own immune cells, won’t be cheap.

By its own reckoning, the UK’s tough watch dog on drug pricing has said that these drugs would be worth up to $649,000 a year, given the young patients it’s targeting first. Analysts, meanwhile, have pegged the price at anywhere from $400,000 to $750,000.

In a call with analysts, Novartis execs said it would keep the price well below the high end, setting the cost at $475,000 for a drug that will be available in 20 centers within one month, and 35 soon after.

In a statement the company said it “is collaborating with CMS to make an outcomes-based approach available to allow for payment only when pediatric and young adult ALL patients respond to Kymriah by the end of the first month. Future potential indications would be reviewed for the most relevant outcomes-based approach.”

Joe Jimenez

Said Joseph Jimenez, CEO of Novartis:

Five years ago, we began collaborating with the University of Pennsylvania and invested in further developing and bringing what we believed would be a paradigm-changing immunocellular therapy to cancer patients in dire need. With the approval of Kymriah, we are once again delivering on our commitment to change the course of cancer care.

This approval marks a major shift in oncology, with a truly revolutionary approach to treating cancer. That hasn’t escaped the notice of big players and small, with a host of developers looking to do much better, more safely, with new drugs in the pipeline.

It’s a big day in the biotech world, but it’s just the official start date for a new era.

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Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT
Director of Operations
Atlas Venture Cambridge, MA

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