"Yescarta, a chimeric antigen receptor (CAR) T cell therapy, is the second gene therapy approved by the FDA and the first for certain types of non-Hodgkin lymphoma", the FDA said in a statement. The treatment will cost almost $375,000, which is $100,000 less than the first FDA-approved CAR-T treatment. In a healthy body, the immune system can recognize abnormal, cancerous cells, but for people with cancer, it doesn't recognize that the cells are spreading.
Gilead shares were up 1.1% at $80.90 Thursday morning, in a 52-week trading range of $63.76 to $86.28 and with a consensus analyst target price of $84.20.
Yescarta, formerly KTE-C19, is a CD19-directed genetically modified autologous T cell immunotherapy that will be manufactured specifically for each individual patient. "In just several decades, gene therapy has gone from being a promising concept to a practical solution to deadly and largely untreatable forms of cancer", said Food and Drug Administration Commissioner Scott Gottlieb in a statement. He called the approval a "milestone". There are about 7,500 patients with refractory DLBCL in the USA annually who are eligible for auto T therapy.
Yescarta uses gene therapy techniques called CAR-T to boost the capacity of T cells, the immune-system cells that cancer can evade.
"Today is an important day for patients with relapsed or refractory large B-cell lymphoma who have run out of options and have been waiting for new treatments that may help them in their fight against cancer", Gilead Sciences President and CEO John Milligan said in a press release (Gilead owns Kite as of 2016). The therapy was evaluated in clinical studies involving more than 100 adults.
The NCI licensed the treatment to Kite Pharma, and once it was shown to cause complete remission in half the patients who tried it, Gilead bought Kite.
The adverse effect profile remains the same for this treatment as for tisagenlecleucel-the drug has a boxed warning for cytokine release syndrome and for neurologic toxicities, which can both be fatal or life-threatening. Other side effects include infections, low blood cell counts, and dampened immune system.
"The FDA is requiring that hospitals and their associated clinics that dispense Yescarta be specially certified".
And as part of the approval, Kite Pharma Inc. must conduct a study of patients who take Yescarta. However, since the eligible patients are facing such a high risk of death, most seem to think it's worth the risk. "That policy will also clarify how we will apply our expedited programs to breakthrough products that use CAR-T cells and other gene therapies". A different type of gene therapy is waiting in the wings at the FDA.
Arie Belldegrun, the Israeli-American founder of Kite Pharma, referred to the promising research when commenting on Gilead's purchase of Kite in August.
Gottlieb added that the FDA "will soon release a comprehensive policy to address how we plan to support the development of cell-based regenerative medicine".
The FDA decision opens the door for a new gene therapy to treat adults with aggressive lymphoma, noted Maloney.