July 26, 2017

Novartis FDA Panel Approval: 'Pharma Innovation At Its Best'

14 July 2017, 12:36 | Elizabeth Houston

FDA advisers back Novartis' game-changing personalized CAR-T for cancer, approval in sight

Novartis dodged deaths from its CAR-T Therapy, but FDA still has safety concerns

The FDA panel made their recommendation after reviewing CTL019's performance in a range of clinical trials including the ELIANA study, which was the first pediatric global CAR-T cell therapy registration trial.

An advisory panel to the Food and Drug Administration unanimously backed approval of Novartis' CAR-T therapy tisagenlecleucel for an aggressive type of leukemia, likely signaling a clear path towards a regulatory OK later this year. In the United States, about 15% of children and young adults with acute leukaemia relapse following treatment.

This treatment has given patients, who were facing death as other treatments failed, long remission periods and possibly even cured them. Compared with standard treatment, which required dozens of spinal taps and painful bone-marrow tests, the T-cell treatment was far easier to tolerate, McMahon said, and he urged the panel to vote for approval.

The Swiss drug company Novartis AG, which developed the treatment, is expected to receive a decision on CTL019 by October from the FDA in October, according to Bloomberg. The treatment requires the extraction of a patient's own infection-fighting T-cells, which are then genetically engineered to recognize and fight the cancer cells affecting the patient, and infused back into the bloodstream.

The National Cancer Institute's James Gulley said in the panel discussion: "This is a product that has shown patient benefit, and I think our goal here is to try and find ways that we can make sure we have optimal safety and potency".

But the FDA advisers did not seem very concerned about the roadblocks and the meeting finished with a unanimous yes vote more than an hour ahead of schedule. Then sensor cells (antigen receptors) that can seek out certain cancers and destroy them are added to the T-cells. Children with cancer are closely monitored for years in clinical practice, said Timothy Cripe, chief of the hematology/oncology division at the Nationwide Children's Hospital.

Other CAR-T therapies are on the horizon, including a promising one from Kite Pharma.

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Close on Novartis' heels is a team from Kite, a CAR-T-focused biotech which had hoped to be the first to the market.

Other panelists called the treatment a "major advance" and that novel therapy meets a "dire, unmet need". But treatment gave their daughter a few extra months of happy life, Kappen said.

But the drug has side effects that can be fatal, such as cytokine release syndrome or CRS, which "looks like sepsis" and causes blood pressure to drop dangerously low, said Diefenbach.

FDA reviewers said ahead of the meeting they were concerned about the potential for these viruses to infect patients and cause secondary cancers.

Each treatment is created for each individual patient by having their cells removed, frozen and shipped to a Novartis plant for processing. The T-cells hone in on a protein called CD-19 that is found on the surface of most B-cells. The deal covers Celyad's rights to U.S. Patent No. 9,181,527 related to allogeneic human primary T cells that are engineered to be T-cell receptor deficient and express a vehicle.

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.

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