Rare Disease Report

Blincyto Gets FDA Full Approval to Treat ALL in Adults and Children

JULY 12, 2017
James Radke
The U.S. Food and Drug Administration (FDA) has granted full approval of Blincyto (blinatumomab) to treat relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL) in adults and children.
ALL is a rare and rapidly progressive cancer. Adults with relapsed or refractory ALL generally have a poor prognosis with a median survival time of 3 to 5 months.
In December 2014, the FDA gave accelerated approval of Blincyto to treat Philadelphia chromosome-negative (Ph-) precursor B-cell ALL, based on impressive complete remission data. The FDA, however, stated at the time that Blincyto’s manufacturer (Amgen) needed to conduct a study to verify that the drug improves overall survival.
The supplemental Biologics License Application (sBLA) submitted showed the single-agent immunotherapy to improve overall survival in a Phase 3, randomized, active-controlled, open-label TOWER study involving 405 adult patients with Ph- relapsed or refractory B-cell precursor ALL. The study published in the New England Journal of Medicine showed that the median overall survival was 7.7 months in the blinatumomab group and 4.0 months in the standard of care chemotherapy group (P = .01). 
According to Amgen, the study was stopped early based on the superior efficacy in the Blincyto group observed in the study.
The sBLA also included data from a Phase 2 single-arm, multicenter, open-label ALCANTARA study in 45 adult patients with relapsed or refractory Ph+ B-cell precursor ALL. A study published in the Journal of Clinical Oncology showed that of the 45 enrolled patients, 16 achieved complete remission (CR) or CR with partial hematologic recovery (CRh) during the first 2 cycles and 88% of CR/CRh responders achieved a complete minimal residual disease response. 
Per Amgen: "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, M.D., 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."
"Relapsed or refractory ALL is often a lethal disease, with a median overall survival of just four months on standard of care chemotherapy," said Bijal D. Shah, M.D., 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 drug has a boxed warning for cytokine release syndrome and neurologic toxicities and is also under a risk evaluation and mitigation strategy (REMS) program in the U.S.

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