Last week a panel of oncology experts summoned by the FDA voted unanimously in favour of Novartis’ groundbreaking CAR-T cancer therapy. This was the first advisory committee for CAR-T therapy and the positive advice is an important milestone for this exciting novel option for cancer patients. An advisory committee is typical in the final stage of the drug-approval process and the outcome tends to correlate well with the final marketing authorization.
Chimeric Antigen Receptor T-cell or CAR-T therapy is a personalized therapy where the patient’s immune cells are harvested and engineered to fight cancer. The patient’s killer T-Cells are equipped with a receptor to detect the cancer and with a stimulating domain to trigger the attack of the cancer cells. The entire process is a true masterpiece of biotechnology and the full production cycle encompasses about three weeks. The results Novartis achieved with this therapy in relapsed/refractory paediatric acute lymphoblastic leukaemia (ALL) are truly spectacular. In the case of the children treated, for whom all therapeutic options were depleted, Novartis CTL019 CAR-T therapy achieved a complete remission in 82% of cases and, after 6 months, 60% of those were still relapse-free. Although the benefit is clear, there is also a debate around the risks of CAR-T therapies. The aggressive immune response induced by CAR-T therapy can lead to severe side effects and has even led to lethal events in competitors’ CAR-T therapies in different cancers. Although caution is always required, one should bear in mind that those patients had no other treatment options left.
The FDA review process for Novartis’ CAR-T therapy in paediatric ALL is now most advanced, with a final FDA decision expected before the end of October. Next up for a regulatory decision is Kite Pharma in lymphoma, with an FDA decision expected in November. Other names to watch in this space with advanced clinical programmes are Juno Therapeutics and Bluebird Bio.
The current unanimous vote from the advisory for Novartis CTL019 CAR-T therapy is a crucial milestone in the development of CAR-T therapy, which has the potential to revolutionize cancer treatment. It is the first time that we have seen FDA feedback for this new treatment and it is very encouraging to see that the expert panel is this positive. There is a direct impact for names such as Juno Therapeutics, Kite Pharma and Bluebird Bio, which have also demonstrated spectacular results with their CAR-T therapy, but this is likely also the first gene therapy ever to be approved by the FDA. It demonstrates the FDA’s commitment to innovative drugs that save patients’ lives. Overall, this acknowledgement for innovative therapies, together with the recent positive evolutions in the drug-pricing discussion, confirm our positive stance on the biotech sector.
With last week’s panel decision, this truly revolutionary way of treating cancers is now close to reaching patients. The Candriam biotech strategy is invested in those innovative CAR-T therapies via holdings in Juno Therapeutics, Kite Pharma and Bluebird Bio.