Novartis gains speedy FDA review for new CAR-T med as Kite race begins

The drug originally came out of work out of the University of Pennsylvania, which Novartis teamed up with in 2012.

Swiss major Novartis is still hoping to be first to market a pioneering new class of oncology medicine known as CAR-T after getting a priority review from the FDA last night for its blood cancer med CTL019.

Novartis is gunning for its first license with CAR-T (more are in the pipeline) in kids and young adults with relapsed or refractory (R/R) pediatric and young adult patients with B-cell acute lymphoblastic leukemia (ALL).

Last year, Novartis came out with new data from its phase 2 test that saw 82% (41 out of 50 patients) achieve complete remission or complete remission with incomplete blood count recovery at three months after an infusion of its med CTL019 infusion in young R/R patients with B-cell ALL.

The Big Pharma is however in a race with Kite Pharma, a specialist CAR-T biotech working on axicabtagene ciloleucel (KTE-C19), which has already kick-started a rolling submission with the FDA for a BLA of its med as a treatment for patients with R/R aggressive B-cell non-Hodgkin lymphoma who are ineligible for autologous stem cell transplant.

The two will likely have a very close battle to go down in the history books as the first to market CAR-T; a priority review means that it could be on the market within 6 months.

If Novartis does turn victor, this will come as a major turnaround given that last year, it announced plans to cut back on its gene and cell therapy unit (which developed this drug) and saw the exodus of many of those who had helped create and nurture it.

The drug originally came out of work out of the University of Pennsylvania, which Novartis teamed up with in 2012.

“With CTL019, Novartis is at the forefront of the science and development of immunocellular therapy as a potential new innovative approach to treating certain cancers where there are limited options,” said Vas Narasimhan, global head of drug development and CMO of Novartis.

“The priority review and file acceptance of CTL019 by the FDA brings us one step closer to delivering this novel treatment option to children and young adults with r/r B-cell ALL in the United States.”

This was also good news for British biotech Oxford BioMedica, which produces the lentiviral vector expressing CTL019 and has a CAR-T partnership with Novartis. It said this morning: “The news that the FDA has accepted the BLA for CTL019 and granted it priority review is an important development for Oxford BioMedica. 

“We continue to work closely with Novartis in delivering the lentiviral vector expressing CTL019, a product described earlier this year by Novartis as having ‘blockbuster’ potential.”

Its blockbuster potential will likely come with a blockbuster price: Analysts at Jefferies believe Kite's Axi-Cell, across all indications, could cost up to $300,000 in the U.S., while in Europe, they’re predicting a price tag of $204,000.

These prices have gone up by around $100,000 and $80,000 respectively in recent months, as new data have come in, and are similar to those price tags believed to be applied to Novartis’ med.

Another CAR-T biotech, Juno, had looked to be alongside Novartis and Kite in the race to market, but is now out of the running after a string of deaths from several studies last year saw it ditch its leading CAR-T med as it had to rely on JCAR017, a med further back in its pipeline.

Bluebird bio is a little further behind in regulatory terms than Novartis and Kite, posting some early but impressive data last year from a phase 1 trial showing its anti-BCMA CAR-T cell product candidate, which was given to patients with R/R multiple myeloma and were heavily pretreated, had an ORR rate of 78%, and with no dose-limiting toxicities and no grade 3 or higher neurotoxicities or CRS coming out of its test.