FDA advisers recommend approval of new cancer therapy

Posted July 14, 2017

The FDA's advisory committee on cancer drugs has offered a group thumbs-up for Novartis' trailblazing CAR-T CTL019 (tisagenlecleucel), marking a key milestone for the pharma giant as it nears the likely launch of a groundbreaking personalized cell therapy for B-cell acute lymphoblastic leukemia.

Kite's therapy also hurt the antibody-producing B cells, but they came back.

"This is a major advance", Dr. Malcolm A. Smith of the National Cancer Institute, told Fox News, saying that the treatment was "ushering in a new era".

"We believe that when this treatment is approved it will save thousands of children's lives around the world", said Tom Whitehead, the father of a 12-year-old who is now cancer-free after receiving CTL019. Much of the early work on CTL-019, now renamed tisagenlecleucel, took place in the university's labs and clinics. The panel today was just a body of panelists to determine if this therapy should be approved.

FDA's clinical reviewer, Dr. Maura O' Leary, noted that the treatment causes an inflammatory collapse called a cytokine storm, a sometimes deadly reaction, that can also cause nerve damage. It had ruled that the benefits of patients receiving this treatment outweigh the overall risks.

Tisagenlecleucel is a type of immunotherapy called chimeric antigen receptor T-cell therapy, or CAR-T. The ELIANA Trial originally enrolled 88 patients with relapsed or refractory B-cell ALL. That is because of the nature of the disease, and not because the treatment is toxic.

Observers crowded into the FDA's presentation room this morning, rubbing shoulders with CAR-T celebs like Penn's Carl June with many, many more looking on online. Still, those patients who were evaluated showed some remarkable progress. A Novartis study of the technique found that 83% of participants achieved remission. What does a 95% confidence interval mean? The primary endpoint of the study was looking for an improvement in complete remission or complete remission with incomplete blood count recovery over time with treatment of CTL019.

Other biotech and pharmaceutical companies are developing types of gene therapy to treat solid cancers and rare gene-linked diseases. The first of which is the safety risks discussed during the advisory panel committee. That means that long-term effects are not yet widely known. Such safety issues could be the possibility of a tumor growth, viral infections and brain toxicity. Penn has a next-generation CAR-T under development, dubbed CTL-119, which is not licensed to Novartis.

The FDA is expected to make a final decision on approval by October 3.

Insurance companies said their decision to cover Novartis's treatment and any follow-up therapy will be based on the clinical results of the drug. The FDA panel recommended that patients who get the treatment be monitored for 15 years.

The news is not only good for Novartis, which expects peak sales of at least $1Bn.

Novartis also proposed a risk evaluation and mitigation strategy to ready sites for using tisagenleleucel.

The FDA is not required to accept the recommendations of its advisory panel, but it usually does. The only downside might be the market for patients with ALL. One possibility is that the agency is anticipating that CAR-T products will be developed for patients who aren't at the end of the line. I am not receiving compensation for it (other than from Seeking Alpha).