UPDATE: Amgen's BiTE Received FDA Priority Review Designation

Amgen

today announced that the U.S. Food and Drug Administration (FDA) has accepted for review the Biologics License Application (BLA) for the investigational bispecific T cell engager (BiTE®) antibody construct, blinatumomab. The BLA is for the treatment of adults with Philadelphia-negative (Ph-) relapsed/refractory B-precursor acute lymphoblastic leukemia (ALL), a rapidly progressing cancer of the blood and bone marrow.1 As part of the acceptance, the FDA granted blinatumomab priority review with a Prescription Drug User Fee Act (PDUFA) action date of May 19, 2015. A Marketing Authorization Application (MAA) has also been submitted to the European Medicines Agency (EMA) via the centralized procedure for approval to market blinatumomab for the treatment of adults with Ph- relapsed/refractory B-precursor ALL. The submissions include data from a Phase 2 trial of adult patients with Ph- relapsed/refractory B-precursor ALL treated with blinatumomab, which met its primary endpoint. "The FDA's acceptance of our BLA submission and designation of priority review for blinatumomab underscores the need to provide new treatment approaches for adult patients with relapsed or refractory ALL, and we are encouraged by the Agency's expedited review," said Sean E. Harper, M.D., executive vice president of Research and Development at Amgen. "Blinatumomab has the potential to make a significant impact for these patients, and this milestone, along with other ongoing filings around the world, represents the potential of BiTE® technology in cancers that are challenging to treat." BiTE® antibody constructs represent an innovative immunotherapy approach that helps the body's immune system target cancer cells. Blinatumomab, the first of the investigational BiTE® antibody constructs, has received orphan drug designation from the EMA and FDA, and breakthrough therapy and priority review designation from the FDA for the treatment of ALL. According to the FDA, priority review designation is assigned to applications for drugs that treat serious conditions and would, if approved, provide significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions. A priority review designation will set a goal date for taking action on an application within six months of receipt. In the U.S., more than 6,000 cases of ALL will be diagnosed in 2014, and in the European Union, it is estimated that more than 7,000 cases of ALL are diagnosed each year.2,3 In adult patients with relapsed or refractory ALL, median overall survival is just three to five months.4