Novartis' leukaemia drug CTL019 has been awarded 'breakthrough' status in the USA, giving the personalised cell therapy a potential fast pass to market approval.
The filing was submitted by the Swiss drug giant's development partner the University of Pennsylvania's Perelman School of Medicine (Penn), and seeks to develop the cancer immunotherapy as a treatment for paediatric and adult patients with relapsed/refractory acute lymphoblastic leukaemia (r/r ALL).
There remains a high unmet need for patients with the condition, and researchers say they are "excited about the strength of the positive early data" reaped from clinical trials thus far.
CTL019 uses CAR technology to reprogram a patient's own T cells to hunt down cancer cells expressing specific proteins called CD19. Once reprogrammed, these T cells (now called CTL019) are re-introduced into the patient's blood, where they proliferate and bind to the targeted cancer cells and destroy them.
Novartis holds the worldwide rights to CARs developed through the collaboration with Penn for all cancer indications, including the lead program CTL019, which is currently being investigated in Phase II clinical trials.
This is the fifth breakthrough therapy designation for Novartis.