The U.S. Food and Drug Administration (FDA) has accepted for review Kite Pharma’s application requesting the approval of its investigational CAR T-cell therapy KTE-X19 for treating adult patients with relapsed or refractory mantle cell lymphoma (MCL).
Kite’s biologics license application (BLA) received priority review status, which shortens review time from the standard 10 months to six months. The designation is granted to therapies with potential to bring significant benefits in safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions. A final decision is expected by Aug. 10.
The European Medicines Agency (EMA) is reviewing a similar application for the use of KTE-X19 in the European Union. The therapy also has received breakthrough therapy status from the FDA and priority medicines (PRIME) designation by the EMA for the same indication.
“Despite recent advances, patients with relapsed/refractory mantle cell lymphoma currently face a significant lack of effective treatment options once their disease no longer responds to currently available therapy,” Ken Takeshita, MD, Kite’s global head of clinical development, said in a press release. “Based on the encouraging results for KTE-X19, we are eager to continue discussions with the FDA on how to bring this innovative treatment to these patients who may benefit from CAR T therapy.”
KTE-X19 is a type of immunotherapy called CAR T-cell therapy, which harnesses a patient’s own immune cells to fight cancer. Specifically, the therapy consists of immune T-cells that are harvested from the patient and genetically engineered in the lab to produce a chimeric antigen receptor (CAR), a man-made receptor that helps immune cells recognize a specific cancer protein, called CD19.
The modified T-cells are then expanded and infused back into the patient for treatment, where they will bind and target the cancer cells for destruction. KTE-X19 has an additional enrichment step in its manufacturing process, which distinguishes it from Kite’s approved therapy Yescarta (axicabtagene ciloleucel), a necessity for some B-cell cancers like MCL.
If approved, KTE-X19 will be the first CAR T-cell therapy for MCL, both in the U.S. and in Europe.
The applications were supported by results from the Phase 2 ZUMA-2 trial (NCT02601313), in which a single administration of KTE-X19 significantly reduced tumor burden in 93% of relapsed or refractory MCL patients. Two thirds of patients achieved a complete response, meaning that their cancer disappeared completely.
The open-label trial included 60 patients who received up to five prior therapies, including a chemotherapy, an anti-CD20 antibody, and a Bruton’s tyrosine kinase inhibitor (BTKi). The median number of prior therapies was four.
At the time of the analysis — after a median follow up of 12.3 months — 78% of patients who had achieved a complete response were still in remission. Also, 12 months after treatment, 83% of participants were still alive and 61% of them showed no signs of disease progression.
The investigational therapy also showed a manageable safety profile, with neurologic events seen in 31% of patients and cytokine release syndrome (CRS, a serious inflammatory response to the therapy) observed in 15%. Two patients died as a result of adverse effects, including an infection possibly related to the treatment.
An expanded access study, ZUMA-18 (NCT04162756), is available for patients with relapsed or refractory MCL, aiming to provide access to KTE-X19 until the therapy is commercially available. Go here for eligibility criteria and here for more information on the trial’s two locations, in New Jersey and Texas.
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