FDA approves Blincyto to treat relapsed or refractory B-cell precursor acute lymphoblastic leukemia

Blincyto is the first and only Bispecific T cell Engager (BiTE) immunotherapy to demonstrate superior overall survival versus standard of care chemotherapy.

"For researchers and physicians, overall survival is the primary goal of treatment and the gold standard of outcomes, demonstrating a clear value to patients," said Anthony Stein, study investigator and co-director of the Gehr Family Center for Leukemia Research, City of Hope, Duarte, Calif. "Data from the TOWER study support the use of this single agent bispecific T cell engager immunotherapy, the first to demonstrate superior overall survival in patients with Philadelphia chromosome-negative relapsed or refractory B-cell precursor ALL, offering a much needed alternative with significantly improved outcomes over standard of care chemotherapy."

Blincyto, the first single-agent immunotherapy to treat patients with Philadelphia chromosome-negative (Ph-) relapsed or refractory B-cell precursor ALL, was previously granted breakthrough therapy designation and accelerated approval. It is also the first-and-only FDA-approved CD19-directed CD3 bispecific T cell engager (BiTE) immunotherapy, and the first bispecific antibody construct from Amgen's BiTE platform.

"Relapsed or refractory ALL is often a lethal disease, with a median overall survival of just 4 months on standard of care chemotherapy," said Bijal D. Shah, medical oncologist, Moffitt Cancer Center, Tampa, Fla. "As a physician, my goal is to identify treatments that improve response rates in patients with aggressive hematologic malignancies. Blincyto is an option that has been shown to help these high-risk patients fight their disease."

The approval is based on results from the TOWER study, which found that Blincyto demonstrated a superior improvement in median OS over standard of care (SOC) chemotherapy, nearly doubling median OS. The study showed that median OS was 7.7 months (95%) for Blincyto versus four months (95%) for SOC. The approval is also based on data from the Phase II ALCANTARA study, which evaluated the efficacy of Blincyto in adult patients with Ph+ relapsed or refractory B-cell precursor ALL.

"We are pleased that the FDA has granted full approval for Blincyto, marking a significant milestone for certain patients with relapsed or refractory ALL," said Sean E. Harper, executive vice president of Research and Development at Amgen. "This approval supports the use of Blincyto in a broader spectrum of patients, including those with few options to date, such as Philadelphia chromosome-positive patients, and reinforces the potential of the BiTE platform as a novel approach to immuno-oncology."