Novartis gets a speedy review for its CAR-T pioneer as FDA lines up a marketing decision

Vasant Narasimhan, Novartis

Novartis $NVS has pulled into the inside track at the FDA with its CAR-T CTL019, picking up a priority review from the agency that will land a marketing decision for relapsed and refractory (r/r) pediatric and young adult patients with B-cell acute lymphoblastic leukemia.

The pioneering application comes as we wait for Kite to make its move at the agency after being forced to wait for 6-month data on its rival CAR-T. Kite $KITE has promised to finish its rolling application by the end of Q1, now just days away. And a spokesperson for Kite tells me now that the biotech is on track to wrap the submission by Friday.

The priority review leaves Novartis in shooting range of the world’s first approval for a CAR-T, which takes T-cells from patients and then engineers them to attack cancer cells. Blood cancers proved to be the best, early targets in the field, which has seen a lineup of companies jump in on the promise of dramatic responses for some patients.

Novartis pushed ahead in CAR-T after stunning the industry with its decision last summer to dissolve its cell and gene therapy unit, a story we broke just after launching Endpoints News. The pharma giant insisted that the sudden sharp turn, which prompted the unit’s chief, Usman “Oz” Azam, to join an exodus of execs out of Novartis over the past year, wouldn’t sidetrack its programs.

But it’s definitely in a tight race for the finish line.

The progress of these early leaders hasn’t come easily. Juno was once in the lead pack, until its frontrunner was sidetracked twice by patient deaths which it never fully explained. Now that lead program has been shelved as Juno switches focus to the next therapy in the pipeline, handing the lead to Novartis and Kite.

Novartis says it’s prepping an EMA application and plans additional filings for CTL019 in the US and EU markets later this year, including a BLA with the FDA for treatment of adults with r/r diffuse large B-cell lymphoma (DLBCL) and applications for marketing authorization with the EMA in r/r B-cell ALL and r/r DLBCL.

Wedbush’s David Nierengarten recently speculated that Kite’s data update put it in line for a late 2017 approval, shifting the focus to pricing. He noted:

“(W)e note to justify the current market capitalization, we would require either a price point of $500,000 per course or up to 50% market share (or some combination), both of which, while possible, we believe is less likely.”

But some analysts start this bidding at $250,000.

Whoever gets onto the market first will have a big edge in setting the price for these first wave drugs.

“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 Chief Medical Officer, 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.”

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Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT
Director of Operations
Atlas Venture Cambridge, MA

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