Generic name: axicabtagene ciloleucel
Company: Kite Pharma
Disease: non-Hodgkin's lymphoma
2022 sales estimate: $1.4 billion
Kite Pharma is hopeful that it can be the first company to bring a CAR-T med to the market in the U.S. later this year, in the form of its blood cancer candidate KTE-C19 (a.k.a. axicabtagene ciloleucel), but it will fight a close battle with Novartis for the honor of being first.
Kite appeared to pull ahead in the race to market last year when rival biotech Juno Therapeutics was hit by a series of deaths in a CAR-T drug trial, and several halts on a key study. It looked as if Novartis, after last year cutting much of its gene and cell therapy team in 2016, was pulling back on its research in the area as well.
However, while Kite kickstarted its rolling submission with the FDA for KTE-C19 in early December 2016, for an indication in relapsed or refractory aggressive B-cell non-Hodgkin's lymphoma (NHL), Novartis was gearing up for its own FDA talks, too.
In the same week in December as Kite, Novartis posted new phase 2 data that “set the stage” for a date between its CAR-T, known as CTL019, and the FDA in “early 2017” for pediatric and young adult patients with r/r B-cell ALL. Kite said it expects to complete its BLA submission by the end of Q1, setting up a tantalizing race to approval, as it “plans to commercially launch KTE-C19 in 2017.”
Novartis said it plans to submit to the EMA this year too, having already received the Agency’s PRIME designation, which can speed up review times. Meanwhile, Kite has the same PRIME tag and European submission plans for 2017. Both companies also hope for a slew of other blood cancer indications for their respective meds in the coming years.
Goldman Sachs has previously said it sees $1.9 billion in sales for KTE-C19 in 2026. Hitting that total will likely depend on its ability—and the ability of its competitor meds—to expand into other indications, including solid tumors, something it has not managed as well as in blood cancers. Price tags will also matter; some analysts have predicted the price of new CAR-Ts at around $300,000 per course.