CAR-T therapies for ALL may encounter FDA flexibility regarding postapproval confirmatory study requirements – experts

26 May 2016

Chimeric antigen receptor T-cell (CAR-T) therapies in acute lymphoblastic leukemia (ALL) may not require randomized confirmatory studies if they win accelerated FDA approval, experts said.

The high complete remission (CR) rates seen in ALL trials of CD19-targeting CAR-Ts from Juno Therapeutics (NASDAQ:JUNO), Kite Pharma (NASDAQ:KITE) and Novartis (VTX:NOVN) may make it unethical to randomize patients to another therapy, they explained. How to design a confirmatory trial is debatable given the magnitude of benefit from CAR-T versus existing therapies and the complexity of designing a randomized study, they noted. The FDA could instead require long-term follow-up data to grant full approval, experts said.

Kite's ZUMA-3 Phase I/II registration-directed study (NCT02614066) of
KTE-C19 is expected to have data next year. Meanwhile, Juno's registrational Phase II trial (NCT02535364) of JCAR015 has a primary completion date of December 2017, though an analyst report anticipated a BLA filing late this year.

Novartis will file for approval of CTL019 in early 2017 for pediatric ALL based on an ongoing Phase II study (NCT02435849), said a spokesperson.

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