The FDA’s recommendation for Novartis’ CTL019 (tisagenlecleucel) opens the way to a new generation of cell-based immunotherapies to treat cancer. 

Yesterday, the FDA’s Oncologic Drugs Advisory Committee (ODAC) held a hearing to evaluate Novartis CTL019. The committee voted unanimously (10-0) in favor of the therapy, which the FDA will certainly weigh in when making the final decision on approval in October. If positive, CTL019 would be the first CAR-T therapy in the market.

The news is not only good for Novartis, which expects peak sales of at least $1Bn. The FDA’s recommendation validates the CAR-T technology many others are developing, including Juno Therapeutics, Cellectis, Celyad, and, most notably, Kite Pharma, which is expecting a decision on the approval of its own CAR-T candidate in November.

Novartis CTL019 FDA recommendation Kite Pharma

The committee has asked Novartis to commit to 15 years of follow-up, given the therapy is following a fast-track approval and no long-term data is available yet. The short-term results, however, have convinced the experts that it’s worth giving CTL019 a try: after 12 months, 79% of patients with acute lymphoblastic leukemia (ALL) were still alive, and 75% had not relapsed again.

The main concerns during the hearing were toxicity and manufacturing. Regarding toxicity, CAR-T trials run by Novartis rivals Kite Pharma and Juno Therapeutics have made headlines in the last months due to several patient deaths caused by neurotoxicity due to the aggressive treatment. Novartis had previously reported severe side effects in 50% of patients, but none regarding neurotoxicity, and one patient that died of cerebral hemorrhage.

Novartis CTL019 FDA recommendation

Regarding manufacturing, the nature of the therapy poses a challenge that doesn’t exist with conventional drugs. Since the therapy is made using the patient’s own immune cells, they need to be sent to specialized facilities where the treatment is manufactured individually. Nine patients on the trial had to be discontinued because CTL019 could not be manufactured for them.

But Novartis is already working in a new generation of CAR-Ts that can be manufactured as an off-the-shelf therapy instead of individually. This would also reduce the price, which is bound to start a controversial debate if the therapy is approved. Last May, Novartis entered a license agreement with the Belgian Celyad to access its allogeneic technology. The French Cellectis and its partner Servier are also developing their own off-the-shelf CAR-T, which was the first to enter clinical trials just last month.


Images via Christoph Burgstedt / Shutterstock; Kite Pharma / Linkedin

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