US, EU regulators grant fast track reviews for Novartis' Kymriah filings

Novartis announced Wednesday that the FDA granted priority review to a filing seeking expanded approval of Kymriah (tisagenlecleucel) for the treatment of adults with relapsed or refractory (r/r) diffuse large B-cell lymphoma (DLBCL) who are ineligible for or relapse after autologous stem cell transplant (ASCT). In addition, the European Medicines Agency granted accelerated assessment to an application for the CAR-T cell therapy for the treatment of children and young adults with r/r B-cell acute lymphoblastic leukaemia (ALL) and for adults with r/r DLBCL who are ineligible for ASCT.

In August last year, Kymriah gained FDA clearance for certain paediatric and young adult patients with ALL, making it the first CAR-T cell therapy approved globally. In October, Novartis submitted an application in the US seeking clearance for the treatment of adults with r/r DLBCL who are ineligible for ASCT, later filing for approval in Europe in the same indication, as well as for the treatment of children and young adults with r/r B-cell ALL.

According to Novartis, the submissions in DLBCL are based on data from the Phase II JULIET study, with updated results presented last month at the American Society of Hematology (ASH) annual meeting showing that Kymriah was associated with an overall response rate (ORR) of 53 percent, with 40 percent achieving a complete response (CR). Results also demonstrated that for 46 patients with at least six months of follow-up from infusion, the ORR was 37 percent with a CR rate of 30 percent, and a partial response rate of 7 percent. Meanwhile, Novartis noted that the filing in r/r B-cell ALL included data from the pivotal Phase II ELIANA study.

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Kymriah, formerly known as CTL019, has been priced at $475 000 in the US for a one-time single administration treatment. The therapy, which utilises a patient's own T cells, uses the 4-1BB costimulatory domain in its chimaeric antigen receptor to enhance cellular expansion and persistence. In 2012, Novartis and the University of Pennsylvania entered into a global collaboration to develop CAR-T cell therapies, including Kymriah.

Novartis indicated that it is planning additional regulatory submissions for Kymriah in paediatric and young adult patients with r/r B-cell ALL and adults with r/r DLBCL outside the US and EU in 2018.

For related analysis, read KOL Views Results: Leading oncologist says CAR-T therapies look more similar than different in DLBCL.

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