The U.S. Food and Drug Administration (FDA) has extended by three months its review of Bristol-Myers Squibb’s investigational CAR T-cell therapy, lisocabtagene maraleucel (liso-cel), as a treatment for adults with relapsed or refractory large B-cell lymphoma after at least two prior therapies, the company announced.
Liso-cel’s new Prescription Drug User Fee Act (PDUFA) action date is now Nov. 16, 2020, instead of August as first planned. PDUFA dates are deadlines for the FDA to announce an approval decision.
The approval request, called a biologics license application (BLA), was taken under priority review by the FDA in February, shortening its expected review time to six months from the usual nine or 10 months. But additional information that was submitted by the company after the filing required a delay in the decision date.
Bristol-Myers Squibb (BMS) will work closely with the FDA to support the continued review of liso-cel.
The application, submitted by Juno Therapeutics, a BMS subsidiary, was supported by data from the Phase 1 trial TRANSCEND NHL 001 (NCT02631044).
This study evaluated the safety and anti-tumor activity of liso-cel in 268 patients with aggressive and hard-to-treat forms of B-cell non-Hodgkin’s lymphoma. These included patients with diffuse large B-cell lymphoma, high-grade lymphoma, primary mediastinal B-cell lymphoma and Grade 3B follicular lymphoma; for all, their disease returned or failed to respond well to other treatments.
Results covering 256 patients showed that liso-cel cleared all detectable traces of cancer (complete response) in more than half of these patients (53%), and partially shrunk tumor volumes in another 20%. Responses were similar across all patient groups and lasted a median of 13.3 months.
Liso-cel is a chimeric antigen receptor (CAR) T-cell therapy. This type of immunotherapy involves collecting a patient’s own immune T-cells, and modifying them to produce a chimeric antigen receptor, or CAR, that targets a specific cancer protein.
This is done to boost the ability of T-cells to identify and efficiently kill cancer cells. Typically, modified cells are expanded to millions, and then infused back to the patient to help fight the tumor.
CAR T-cells in liso-cel specifically recognize CD19, a cell surface protein abundant on normal and malignant B-cells.
Liso-cel has been granted breakthrough therapy and regenerative medicine advanced therapy for aggressive relapsing or refractory B-cell NHL by the FDA. The European Medicines Agency gave liso-cel access to its priority medicines program for relapsing or refractory diffuse large B-cell lymphoma.
