Novartis crosses FDA finish line with monumental CAR-T leukemia approval

In a historic day for cancer treatment and the larger biopharma industry, Novartis notched an FDA approval for its CAR-T treatment tisagenlecleucel, a first-ever cell therapy for a rare form of acute lymphoblastic leukemia.

The road to approval for CAR-T has been filled with uncertainty, but Novartis and lead rival Kite Pharma made strides over the last year, notably with Novartis’ recent unanimous FDA panel vote in favor of approval and Kite’s $11.9 billion acquisition this week by Gilead Sciences.

To be sold as Kymriah at a price of $475,000, the Novartis medication is made of re-engineered T cells collected from each patient, which are then infused back into the patient to attack cancer. It will be targeted at children and young adult patients. The cost has already been a matter of intense public debate as the breakthrough technology meets the reality of healthcare spending.

In the agency's approval announcement, new FDA commissioner Scott Gottlieb said the medical community is "entering a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack a deadly cancer."

This FDA approval for Kymriah came well ahead of the medication’s October action date at the agency. Back in June, 10 experts on an agency panel supported its advance. Following the vote, one panelist said the CAR-T med was among the most exciting treatments he’s seen in his lifetime. At the Novartis FDA panel, parents told their children's stories and pleaded with the committee to recommend the med.

Novartis will work with the Centers for Medicare and Medicaid Services for outcomes-based pricing, the drugmaker announced during a conference call after the approval came through. Under that arrangement, Novartis would only collect payments from CMS when patients respond to treatment by the end of the first month.

During the call, the company unveiled a price of $475,000 for a one-time treatment in the approved indication, on the lower end of analyst speculation about the cost. Novartis Oncology chief Bruno Strigini said the price will "support sustainability … while allowing a return on our investment." The company will also introduce a patient access program for patients who can't secure insurance coverage.

Twenty treatment centers will support the launch initially, with the number growing to 32 by the end of the year, execs said Wednesday. Some centers will begin harvesting patients' T cells in the next 3 to 5 days. The company has been "working with payers for a long time," Novartis’ U.S. oncology head Bill Hinshaw said on the call, adding that the company anticipates "excellent coverage" based on those discussions.

Novartis execs also explained that they’ve interacted with health assessment authorities who gave a value range of $600,000 to $750,000 and that they are "pricing below the standard of care" for the condition, referencing the high cost of stem cell transplants.

For its part, Kite’s action date at the agency with its lead CAR-T medication, Axi-Cel, is in November. Executives at that company previously disclosed that the FDA won’t convene an expert panel, signaling growing confidence in the technology.

Of course, plenty of questions remain as CAR-T use is just ramping up, but leaders in the field have made progress with manufacturing and logistical problems lately, as well as safety, which previously plagued Juno Therapeutics.

Cost is one key factor, with activists getting out ahead of the issue even before Novartis and Kite—now Gilead with Monday’s acquisition—announced their prices or indeed won drug approval. Pharma critics including KEI director Jamie Love and Patients for Affordable Drugs President David Mitchell have said that, because taxpayers funded early research, the public should be guaranteed affordable pricing.

But because patient pools are small, manufacturing is complex and the drugs bring a great deal of innovation to the table, analysts expect the drug class to rank among the world’s highest. Market-watchers had floated prices of $400,000 to $750,000 in recent months.

Following a "disappointing" meeting with Novartis leadership, Mitchell said on Tuesday he believes CAR-T will be among the world’s most expensive therapies.