A key FDA advisory committee voted unanimously on Wednesday to recommend approval of Novartis (NVS -0.52%) ground-breaking drug for pediatric and young adult B-cell acute lymphoblastic leukemia, or ALL. The recommendation clears the way for a formal FDA green light, and it begins a new era in the fight against cancer.

A new approach

Chimeric antigen receptor T-cell therapy, or CAR-T, is a new approach involving the re-engineering of a patient's T-cells so that they can better find and destroy cancer cells. T-cells are collected from a patient, sent off to a facility where they are altered, and then they're infused back into the patient.

Scientists work together in a lab on new medicine.

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Novartis CAR-T, tisagenlecleucel, works by reprogramming T-cells to recognize CD19, a protein that's expressed in B-cell cancers, including ALL. However, T-cells can be reprogrammed to spot other proteins found on cancer cells too. Novartis, Kite Pharma, (which has a CAR-T awaiting FDA review for use in non-Hodgkin lymphoma), Juno Therapeutics, and bluebird bio are at the forefront of developing this technology.

Does it work?

On Wednesday, tisagenlecleucel became the first CAR-T to be considered by the FDA's oncology advisory committee, a group of cancer experts tasked with evaluating the efficacy and safety of cancer treatments. Based on their 10 to 0 vote to recommend tisagenlecleucel for approval, it seems they were impressed.

The committee based its vote on results from a 63 patient phase 2 trial of tisagenlecleucel in ALL. In this trial, it delivered an overall remission rate (ORR) of 82.5% at the three-month mark. That's undeniably good, particularly when you consider that the ORR for Amgen's (AMGN -0.64%) Blincyto, which is already approved for use in this patient population, was 44%.

The committee also debated safety risks associated with tisagenlecleucel, including cytokine response syndrome (CRS) and neurotoxicity. Although CRS and neurotoxicity occurred in 47% and 44% of patients in this trial, respectively, the committee's vote shows they believe that safety risks can be managed, and that tisagenlecleucel's efficacy outweighs its risk.

What it means for investors?

The addressable patient population of pediatric and young adult ALL patients isn't very big, but an approval could transform patient treatment, and clear a path for CAR-T's to win approval for use in more common cancers.

There are only 3,100 new cases of pediatric ALL diagnosed in the U.S. annually, and 80% to 85% of these cases are of B-cell origin. Currently, chemotherapy, radiation, or stem-cell transplant are standard of care in ALL. These approaches can be effective, but remission periods can be short, and survival rates can be poor, particularly in advanced cases. Given there's a big need for new treatments that improve outcomes, tisagenlecleucel could become a valuable weapon in a doctor's arsenal.

Novartis' hasn't said how much tisagenlecleucel will cost, but complex cancer drugs can cost $10,000 per month, or more. For example, Blincyto launched with a $178,000 annual price tag when it won FDA approval, and its global sales were $34 million in the first quarter, up 26% year over year.

What's next

Now that the advisory committee has weighed in, all eyes shift to the FDA, which could grant an official OK at any point. Given the high remission rates in its trial, and the number of other CAR-T's that are making their way to the FDA, a tisagenlecleucel's green light would be very good news for patients eager for new treatment options, and very bad news for cancer cells.