FDA Approves New Cancer Treatment

US approves first gene therapy treatment

Analyst John Newman of Canaccord takes yesterday's FDA approval of Novartis' chimeric antigen receptor T-cell (CAR-T) therapy Kymriah as a meaningful victory for Kite Pharma Inc's (NASDAQ:KITE) fellow CAR-T therapy candidate Axi-cel.

"We're entering a new frontier in medical innovation with the ability to reprogram a patient's own cells to attack a deadly cancer", said FDA Commissioner Scott Gottlieb.

An FDA advisory committee had recommended the therapy for approval in July to treat the relapse of a blood cancer known as B-cell acute lymphoblastic leukemia, or ALL. While this was a groundbreaking approval, the therapy will cost $475,000 for 1 treatment, according to Kaiser Health News.

The CAR-T cell treatment developed by Novartis and the University of Pennsylvania is the first type of gene therapy to hit the USA market - and one in a powerful but expensive wave of custom-made "living drugs" being tested against blood cancers and some other tumors, too.

Most patients with ALL recover through other treatments such as radiation, chemotherapy and stem cells. The FDA said hospitals and clinics must become certified to distribute the treatment, meaning they are prepared to recognize and treat CRS and other potentially fatal neurological events.

Each dose of Kymriah contains a patient's own immune cells, which are sent to a lab to be genetically modified using a virus. Once re-injected into the patient's body, the T cells modified to replicate quickly, and the remission is rapid.

The therapy is being licensed and brought to market by the pharmaceutical company Novartis.

"Novartis is collaborating with (Centers for Medicaid Services) to make an outcomes-based approach available to allow for payment only when pediatric and young adult ALL patients respond to Kymriah by the end of the first month".

The price of the therapy is tagged at around $500,000. An immune overreaction called "cytokine release syndrome" can trigger high fevers, plummeting blood pressure and in severe cases organ damage, requiring special care to tamp down those symptoms without blocking the cancer attack. Because Kymriah can have life-threatening side effects, including unsafe drops in blood pressure, the FDA is requiring that hospitals and doctors be specially trained and certified to administer it, and that they stock a certain drug needed to quell severe reactions.

For now, it is only approved for use on ALL.

Dr. Len Lichtenfeld, deputy chief medical officer of the American Cancer Society, is excited by the drug's approval, but he has some reservations. This is elegant science. "There is still much more work to do", Lichtenfeld told Healthline. The safety and effectiveness of Kymriah was tested in one small clinical trial of 63 children and young adults with relapsed or refractory B-cell precursor ALL. Even then, it is prescribed only as a last resort when other forms of treatment have failed.

Still, "a far higher percentage of patients go into remission with this therapy than anything else we've seen to date with relapsed leukemia", said Dr. Ted Laetsch of the University of Texas Southwestern Medical Center, one of the study sites.



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