Novartis’ CTL019 has taken a giant leap towards the finishing line in the race to get the first cancer killing CAR-T cell therapy on the market having won the support of an FDA advisory panel.
The FDA’s Oncologic Drugs Advisory Committee (ODAC) unanimously (10-0) recommended approval of CTL019 (tisagenlecleucel) for the treatment of relapsed or refractory (r/r) paediatric and young adult patients with B-cell acute lymphoblastic leukaemia (ALL).
"The panel's unanimous recommendation in favour of CTL019 moves us closer to potentially delivering the first-ever commercially approved CAR-T cell therapy to patients in need," said Bruno Strigini, chief executive of Novartis Oncology.
"We're very proud to be expanding new frontiers in cancer treatment by advancing immunocellular therapy for children and young adults with r/r B-cell ALL and other critically ill patients who have limited options."
ALL accounts for around 25 percent of cancer diagnoses among children under 15 years old and is the most common childhood cancer in the US. Effective treatment options are limited, and in paediatric and young adult patients with B-cell ALL that have relapsed multiple times or become refractory to treatment, the five-year disease-free survival is less than 10-30 percent.
CAR-T offers a new treatment approach in that it is specifically manufactured for each individual patient. During the process, T cells are drawn from a patient's blood and reprogrammed in the laboratory to create T cells that are genetically coded to hunt the patient's cancer cells and other B-cells expressing a particular antigen.
Novartis’ filing contains data - presented at the American Society of Haematology meeting in December 2016 - from a Phase II study in which 82 percent (41 of 50) of patients infused with CAR-T cells achieved complete remission or complete remission with incomplete blood count recovery at three months post CTL019 infusion.
The submission is currently being given a priority review by the FDA, which is expected to make a decision by October 3. A decision on KITE Pharma’s rival CAR-T, for which approval is being sought to treat B-cell non-Hodgkin lymphoma, is expected in November.
Novartis picked up rights to CTL019 under an agreement with the University of Pennsylvania in 2012, which also gives it worldwide rights to CAR-Ts developed in all cancer indications.
News of ODAC support for the therapy will also be welcomed by the UK’s Oxford BioMedica, which is the sole manufacturer of the lentiviral vector that encodes CTL019, placing it in line for future royalties.