Today, the US Food and Drug Administration (FDA) will convene the Oncologic Drugs Advisory Committee to review Novartis’ personalised cancer therapy, CTL019. The first discussion point of the meeting will focus on manufacturing and product quality control.
Review meeting
CTL019 (tisagenlecleucel) for relapsed and refractory (r/r) paediatric and young adult patients with B-cell acute lymphoblastic leukaemia (ALL) was granted priority review status by the FDA back in March this year. This designation was granted as a result of the outcomes gained from the company sponsored ELIANA study and submission was supported by the findings from a US multicentre trial and an earlier single site trial led by the Children’s Hospital of Philadelphia.
First developed by the University of Pennsylvania, CTL019 is an investigational chimeric antigen receptor T cell (CAR-T) therapy. CAR-T cells are genetically modified antigen-specific immunotherapies, reprogrammed to specifically target cells expressing a disease associated-antigen (in this case CD19). Novartis and Penn entered into an exclusive global collaboration in 2012 for the research, development and commercialisation of this therapy for the treatment of cancer.
As these cells are manufactured for individual patients using their own cells questions have been raised by the FDA concerning the control of product quality through manufacturing processes, as laid out in the background information from the regulatory body. This consideration has been listed as ‘Discussion Point 1’ for today’s review with safety and efficacy of the therapy playing second fiddle.
This is an unusual tact for the FDA, which is generally more focused on whether or not a new treatment works rather than the manufacturing process.
Once the committee meeting has closed and all discussion points reviewed, an independent panel of experts will decide if the therapy is approved.