Waiting Lists are Already Forming for Novartis AG and Kite Pharma's Not-Yet Approved CAR-T Drugs

July 7, 2017
By Alex Keown, BioSpace.com Breaking News Staff

NEW YORK – As the promise of CAR-T therapies becomes closer and closer, consumers are taking notice and lining up for the revolutionary cancer treatments that will soon be available from companies like Kite Pharma and Novartis AG .

But before the treatments can make it to patients, there are some logistical concerns that must be addressed. Unlike many other medications, CAR-T treatments are not able to be mass produced and stockpiled. The treatment is a lengthy process that involves reengineering a patient’s cells in a laboratory before they are then administered back into the patient to fight his or her cancer. Because it’s a lengthy process, Bloomberg noted that many cancer patients may not live long enough to receive a CAR-T treatment. That timeline will force some physicians to “play God” and choose which patients can and cannot receive the CAR-T treatments, Bloomberg said.

Other challenges the two drugmakers will face once their treatments receive clearance from the U.S. Food and Drug Administration (FDA) include some potential deadly side effects, such as cytokine release syndrome that can lead to cerebral edema, and the small number of medical centers across the country that are able to handle CAR-T treatments. Bloomberg said there are less than 50 such centers currently in the United States. When the treatments are approved, Bloomberg said Novartis will begin with 30 to 35 accredited CAR-T sites, while Kite will begin with about 10.

Chimeric Antigen Receptor T-Cell Therapies (CAR-T) are engineered in a laboratory to recognize a specific antigen in a cell and then administered into a cancer patient. The CAR-T cells should, if all goes as planned, multiply within the body and target the antigen and eliminate the threat.

In May, Kite received priority review from the FDA for its CAR-T treatment, axicabtagene ciloleucel. The FDA is set to make a decision on Nov. 29 for the treatment of patients with refractory aggressive non-Hodgkin lymphoma (NHL). As the FDA decision inches closer, Bloomberg reported there are about 7,400 patients in the United States lining up for the new therapy.

While Kite awaits a November decision, Swiss giant Novartis will be before a FDA advisory committee on July 12 for review of its CAR-T therapy, CTL019 (tisagenlecleucel-T), which is aimed at the treatment of pediatric and young adults with relapsed/refractory B-cell acute lymphoblastic leukemia. If Novartis’ treatment makes it through the advisory committee, the FDA is expected to make a final decision by late September. As Novartis stands, during clinical trials the company produced about 250 CAR-T treatments, Bloomberg said. As the company closes in on regulatory approval, Novartis told Bloomberg it is “taking the necessary steps to scale up” production. Citing a June investor’s call, Bloomberg said Novartis’ CAR-T treatment will target approximately 49,000 global non-Hodgkin lymphoma patients.

While Novartis and Kite are making the necessary adjustments for large-scale manufacturing of the CAR-T treatments, Bloomberg pointed out that during clinical trials the delay in making the treatments has cost lives. Last month Novartis reported in a six-month data follow-up of a CTL019 trial of pediatric and young adult patients with B-cell acute lymphoblastic leukemia (ALL) that some patients were unable to receive treatment due to manufacturing issues. Of the 88 patients in the trial, 16 patients did not receive infusions of the drug. Of those, seven were a result of insufficiently formulated CAR-T cell product, Novartis said. Bloomberg pointed to another Novartis CAR-T trial where nine patients did not receive infusions of the treatment due to an inability to reengineer the cells.