Gilead Sciences immunotherapy Yescarta has won FDA approval for follicular lymphoma, providing a new treatment option for patients who have some of the toughest cases of this cancer of the white blood cells.
The FDA decision issued late Friday covers adults whose disease has relapsed or has not responded to two earlier lines of treatments. It’s the third regulatory nod for a chimeric antigen receptor T cell (CAR-T) treatment from Kite, a subsidiary of Foster City, California-based Gilead.
Follicular lymphoma is an indolent, or slow-growing, form of non-Hodgkin lymphoma. Despite growing slowly, this cancer can become aggressive over time. According to Gilead, follicular lymphoma accounts for about 22% of all lymphomas diagnosed globally, making it the most common form of indolent lymphoma.
Kite makes Yescarta from a patient’s own T cells. Those cells are engineered in a lab with genetic modifications that direct those cells to target CD19, a protein on the surface of cancer cells. The modified T cells are multiplied, then infused back into the patient.
The FDA based its approval on the results of a single-arm, open-label study Phase 2 study enrolling 146 patients who have received at least two earlier lines of therapy for their follicular lymphoma. The company said that 91% of patients responded to treatment with the CAR-T therapy. Cancer in an estimated 74% of those patients remained in remission after 18 months.
Gilead reported adverse events grade 3 or higher (severe, but not life threatening) that included an immune response called cytokine release syndrome occurring in 8% of patients. Neurologic toxicity was observed in 21% of patients. Other serious side effects included fever during a period when levels of neutrophils, a type of white blood cell, were abnormally low; encephalopathy; and infections from unspecified pathogens.
Cytokine release syndrome and neurological toxicity are known risks of CAR-T therapies and like earlier approvals of this type of drug, Yescarta’s drug label for follicular lymphoma carries a boxed warning that flags these dangers. The approval requires Gilead to offer a program that informs physicians and patients about these risks and mitigates them. Gilead said that Yescarta use in follicular lymphoma will be covered by an existing risk mitigation program for that drug and another CAR-T therapy, Tecartus.
Yescarta was initially developed by Kite Pharma, which Gilead acquired in a $12 billion deal in 2017. Later that year, Yescarta won its first FDA approval for certain types of non-Hodgkin lymphoma that has not responded to two earlier cancer treatments. That decision followed the first FDA approval of a CAR-T therapy, Novartis’s Kymriah. Gilead’s Tecartus was approved last July as a treatment for mantle cell lymphoma.
The latest Yescarta regulatory nod is an accelerated approval. Retaining that approval status will require Gilead to confirm the drug’s benefit by conducting additional clinical studies.
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