Racing toward a historic CAR-T approval, Novartis wins unanimous FDA panel backing

All eyes in the drug industry turned to the FDA’s Oncologic Drugs Advisory Committee meeting Wednesday, where Novartis won crucial backing for its lead CAR-T drug.

By a 10-0 vote, FDA panel experts endorsed tisagenlecleucel-T to treat patients aged 3 to 25 with relapsed/refractory B-cell acute lymphoblastic leukemia (ALL). The FDA doesn’t have to follow its committee recommendations but typically does. The FDA’s action date for the drug is set for October.

Following the vote, one panelist said it was among the most exciting treatments he’s seen in his lifetime. Another called it a “major advance” that’s “ushering in a new era of treatment."

In phase 2 data submitted to the agency, 82% of patients who received an infusion with the drug, also known as CTL019, achieved complete remission or complete remission with incomplete blood count recovery after three months. Forty-eight percent experienced grade 3 or 4 cytokine release syndrome, though there were no deaths in the study from the complication.

Ahead of the meeting, FDA reviewers stressed that the committee should focus on safety for the med that’s part of an entirely new class of individualized treatments manufactured from a patient's own T cells. After multiple patient deaths, Novartis' rival Juno Therapeutics had to scrap its lead CAR-T program back in March. Kite Pharma reported a death from cerebral edema in May but is pressing ahead with its axicabtagene ciloleucel.

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During a presentation to panelists, Novartis global oncology development head Samit Hirawat said there are more than 600 pediatric and young adult relapsed/refractory B-cell ALL patients who could potentially benefit from the drug. Currently, their treatment options are limited and median overall survival is 3 to 9 months. Tisagenlecleucel-T offers a “new hope,” he said

If the med is approved, Novartis representatives said the company plans to manage a patient registry to track safety over the long run.

In the public comment portion of the meeting, Dr. Megan Polanin, senior fellow at the National Center for Health Research, said the drug didn’t have sufficient supporting data because Novartis only submitted a single study with three months of findings. She said the FDA should at least wait for three more months of data, though she stated she understands the reluctance to wait any longer.

The group also heard from Don McMahon, a parent who told his son Connor’s story and the dramatic improvement he saw on the Novartis CAR-T treatment. McMahon urged the committee to recommend the drug for the sake of U.S. pediatric cancer patients and others around the world; he was one of a number of parents who told their children's stories and pleaded with the committee to recommend the med.

"There are parents watching all over the world who are waiting to hear that this treatment will be available before their child dies of cancer," one parent said.

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Aside from relapsed/refractory ALL, Novartis is testing its CAR-T med in other blood cancers. Kite has applied to the FDA for axicabtagene ciloleucel in refractory non-Hodgkin's lymphoma, with an FDA action date of Nov. 29, according to BioPharmCatalyst.

After the meeting, Leerink Partners analysts wrote that the vote represents a “regulatory de-risking step for the CAR-T group,” including Novartis’ rivals in the field. The analysts felt the meeting was “characterized by an overall very constructive and positive discussion,” with committee members taking note of a “strong efficacy signal achieved in an area of unmet need.”

In a Wednesday statement, Novartis said it has “long believed in the potential of chimeric antigen receptor T cell (CAR-T) therapies to change the cancer treatment paradigm," further thanking the committee for its recommendation and extending its "deepest gratitude" to clinical trial participants and their families.

Whenever CAR-T drugs do reach the market, they’re sure to be expensive. An analysis by U.K. cost watchdog NICE back in March found that they could be worth up to $649,000 in childhood leukemia. Novartis’ R&D chief Vas Narasimhan told Bloomberg last month the company is looking at stem cell transplants as a potential benchmark as well as weighing pay-for-performance deals to help fund the treatments.

A Novartis spokesperson previously said "it is too early for us to comment on pricing; however, we will at the time of approval."