Novartis gets second CAR-T candidate FDA ‘breakthrough’ tag

The BTD is for Novartis' much-anticipated JULIET trial data.

As the battle between Swiss major Novartis and biotech rival Kite Pharma heats up to be the first to market a CAR-T cancer med, the Big Pharma has been handed its second FDA "breakthrough" tag for its eagerly awaited JULIET trial.

This latest label, which can speed things along for Novartis’ CTL019 (tisagenlecleucel), is for relapsed/refractory (r/r) diffuse large B-cell lymphoma (DLBCL). This is the most common form of lymphoma and accounts for about 30% of all non-Hodgkin lymphoma cases.

The drug already has a breakthrough tag for r/r B-cell acute lymphoblastic leukemia (ALL) in pediatric and young adult patients, and was given a priority review at the end of March.

Novartis is in a race with Kite Pharma, a specialist CAR-T biotech working on axicabtagene ciloleucel (KTE-C19), which a day after Novartis got its priority review said it had finished its rolling submission with the FDA for a BLA of its med as a treatment for patients with r/r B-cell non-Hodgkin lymphoma who are ineligible for autologous stem cell transplant.

Kite also has breakthrough labels for DLBCL, transformed follicular lymphoma TFL and primary mediastinal B-cell lymphoma PMBCL.

The two will likely have a very close battle to go down in the history books as the first to market CAR-T.

Novartis says its latest breakthrough designation is based on highly anticipated data from its phase 2 JULIET study, which is looking at the efficacy and safety of CTL019 in adult patients with r/r DLBCL.

Investors have been chomping at the bit for the JULIET data, but Novartis only said they “are expected to be presented at an upcoming medical congress.” Kite Pharma and its investors will also be keen to see how positive, or not, the JULIET data turn out to be.

“At Novartis, we are eager to unlock the full potential of CTL019, including the potential to help patients with r/r DLBCL,” said Vas Narasimhan, global head of drug development and CMO at Novartis.

“We look forward to working closely with the FDA to help bring this potential new treatment option to patients as soon as possible.”

If Novartis does turn victor in the race to market, 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 from the University of Pennsylvania, which Novartis teamed up with in 2012.

Kite's shares were down just over 1% this morning on the news, while Novartis, which also announced a tie-up with Allergan for a NASH combo trial, was 0.3% in the red.