"If approved, it will be the first-ever true gene therapy treatment made available to the USA population and will help accelerate the speed at which we will see even more gene-based therapies come to fruition".
The first part of the advisory committee meeting revolved around manufacturing consistency and quality in producing what Novartis and the FDA described as a "living drug" with each batch representing hope for a unique, distinct patient with relapsed/refractory B-cell acute lymphoblastic leukemia (ALL) - and producing it in time for that child or young adult.
The treatment will be suitable for young people who have not been able to improve on conventional treatments, or those who have suffered a relapse that has caused the recurrence of leukemia after complete recovery, according to the report. The company is planning to maintain a patient registry to help monitor patients who receive the therapy. The Novartis drug is the first CAR-T to be reviewed by the regulator and this and other CAR-T therapies are likely to change the way serious blood cancers are treated. Advisory panel recommendations are not binding on the FDA but the agency typically adheres to what the group urges.
A group of cancer experts advising the FDA has asked the agency to approve a Novartis AG (ADR) (NYSE:NVS) leukemia drug now going by the name CTL019.
The Food and Drug Administration advisory panel has voted in favor of this new gene treatment on which the members voted 10-0.
The immune system-altering treatment is for patients aged 3 to 25 with a blood cancer called acute lymphoblastic leukemia (ALL) - the most common form of childhood cancer in the US.
If approved, tisagenlecluecel would be the first CAR-T therapy cleared for commercial use anywhere in the world, putting Novartis ahead of rivals like Kite Pharmaceuticals and on track to meet its lofty expectations for the treatment. The new treatment allows reprogrammed T cells to create an army of cells that can recognize and destroy cancer after injecting them back into the patient.
Commenting on the treatment, Dr. Bryan said, "Particularly, we are concerned that the same safety and benefit seen in clinical trials may not translate into routine use". The CAR-T space should start to heat up once other companies start to win FDA approval for their CAR-T therapies.
The two significant concerns about the treatment are its severe side effects, which is some cases could be life-threatening, and the risk of secondary cancer from the re-infused cells in the long-term. To generate a batch of tisagenlecleucel, white blood cells are purified from a sample of a patient's blood and shipped to a central processing centre. Experts say the approval will pave the way for a new era of gene-based medicines, which has shown promise in treating a wide range of diseases.
The gene-tweaked T-cells target a protein called CD-19, found on the surface of most B-cells. Of the 52 patients examined, 83% had complete remission, although a lot of them suffered serious side effects.
The global CAR-T market is estimated to hit $8.5 billion by 2028, according to a report released in February by Coherent Market Insights.
In May, Novartis signaled its interest in also developing allogeneic auto T-cell therapies, when it entered a nonexclusive license agreement with Celyad to use its USA patents to produce allogeneic vehicle T cells, in a deal that could generate up to $96 million in up-front and milestone payments for the Belgian biotech.