Vas­ant Narasimhan

There’s been con­sid­er­able spec­u­la­tion of late about the da­ta No­var­tis has col­lect­ed from its JULI­ET study in­volv­ing its close­ly-watched CAR-T drug. The phar­ma gi­ant isn’t the best at com­mu­ni­cat­ing with the out­side world and won’t say what it has seen so far or when ex­act­ly it will be put on dis­play. But they have qui­et­ly giv­en the FDA a peek, and reg­u­la­tors seem im­pressed.

In fact, the FDA has now hand­ed over their break­through ther­a­py des­ig­na­tion for CTL019 as a new ther­a­py for dif­fuse large B-cell lym­phoma.

What’s the big deal?

BTDs are in­creas­ing­ly com­mon, but this one is a bit dif­fer­ent than your run-of-the-mill open door promise at the agency. While No­var­tis and Kite have been run­ning neck and neck to get the first pi­o­neer­ing ap­proval for a CAR-T, No­var­tis is tak­ing its first shot at chil­dren with acute lym­phoblas­tic leukemia while Kite shoots for DL­B­CL. Kite has put its DL­B­CL da­ta up for the world to see, at 3 and 6 months, and now CAR-T ob­servers want to see how they match up in the com­ing ri­val­ry.

The FDA’s BTD in­di­cates that No­var­tis has a good shot at it, but a tac­it en­dorse­ment like this is no guar­an­tee that CTL019 is any bet­ter than KTE-C19, al­so known as axi-cel. Af­ter all, the FDA al­ready hand­ed out break­through sta­tus to Kite’s drug for DL­B­CL, trans­formed fol­lic­u­lar lym­phoma and pri­ma­ry me­di­asti­nal b-cell lym­phoma.

We’ll have to wait for an un­spec­i­fied sci­en­tif­ic con­fer­ence to find out what No­var­tis has been keep­ing se­cret.

In the mean­time Juno has been re­or­ga­niz­ing its CAR-T R&D group, a sto­ry End­points News broke on Sun­day evening, with the hire of Sunil Agar­w­al as the new head of re­search. Juno had to shelve its lead pro­gram af­ter it killed 5 pa­tients in clin­i­cal tri­als. The biotech has fall­en more than a year be­hind the two lead­ers in the field.

Forbes‘ Matt Her­p­er (left) with Sean Park­er and Dr. Carl June (right) – screen­shot

“We are en­cour­aged by the FDA’s recog­ni­tion in the po­ten­tial of CTL019 for this in­di­ca­tion, which fol­lows our promis­ing stud­ies of this ther­a­py for ALL and the FDA fil­ing by No­var­tis in pe­di­atric and young adult ALL that re­ceived pri­or­i­ty re­view,” said Penn’s Carl June, di­rec­tor of the Cen­ter for Cel­lu­lar Im­munother­a­pies at the uni­ver­si­ty’s Perel­man School of Med­i­cine.

The FDA’s Oncology Center of Excellence has been a bright spot within the agency in terms of speeding new treatments to patients. That flexibility was on full display this morning as FDA released new draft guidance spelling out exactly how oncology drug developers can fulfill both the accelerated and full approval’s requirements with just a single randomized controlled trial.

While Congress recently passed legislation that will allow FDA to require confirmatory trials to be recruiting and ongoing prior to granting an accelerated approval, the agency is now making clear that the initial trial used to win the AA, if designed appropriately, can also serve as the trial for converting the accelerated approval into a full approval.

US regulators cleared an ultra-rare drug from Pharming Group, by way of Novartis, on Friday afternoon.

The Dutch biotech said the FDA greenlit leniolisib for an immunodeficiency disease known as activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome, or APDS. People 12 years and older can receive the oral drug, to be marketed as Joenja, beginning early next month, Pharming said, five days ahead of the decision deadline set by the FDA as part of a priority review.