This application represents the first chimeric antigen receptor (CAR) T-cell therapy submitted to the EMA. Axicabtagene ciloleucel is currently under review by the U.S. Food and Drug Administration (FDA), and the FDA has set a Prescription Drug User Fee Act (PDUFA) action date of November 29, 2017.
The MAA for axicabtagene ciloleucel is supported by data from the ZUMA-1 trial, which met the primary endpoint of objective response rate (ORR), with 82 percent (p < 0.0001) of patients achieving a response after a single infusion of axicabtagene ciloleucel. At a median follow-up of 8.7 months, 44 percent of patients were in ongoing response, which included 39 percent of patients in complete response (CR).
The most common Grade 3 or higher adverse events included cytokine release syndrome and neurologic events, which were generally reversible.
“The MAA submission of axicabtagene ciloleucel marks an important global milestone in the development of engineered T cell therapy,” said Arie Belldegrun, M.D., FACS, Chairman, President, and Chief Executive Officer of Kite. “We are excited to work closely with the EMA, Committee for Medicinal Products for Human Use (CHMP) and Committee for Advanced Therapies (CAT) to help bring this potentially transformative therapy to patients in the EU.”
Non-Hodgkin lymphoma (NHL) is a type of blood cancer that affects around 93,000 people in Europe every year.i DLBCL is one of the subtypes of NHL that is aggressive or fast growing.ii While many patients can achieve and maintain complete remission after initial treatment, patients who experience relapse or do not respond to initial treatment historically have poor outcomes.
The company estimates that approximately 7,800 patients in the EU 5 alone may benefit from CAR-T therapy.
In May 2016, the CHMP and CAT granted access to its newly established Priority Medicines (PRIME) regulatory initiative for axicabtagene ciloleucel in the treatment of patients with refractory DLBCL.
Access to the PRIME initiative is granted by the EMA to support the development and accelerate the review of new therapies to treat patients with a high unmet need.