October 10, 2014
By Jessica Wilson, BioSpace.com Breaking News Staff
Amgen , the Thousand Oaks, Calif.-based biopharmaceutical company, yesterday announced that the U.S. Food and Drug Administration (FDA) has accepted for review the Biologics License Application (BLA) for blinatumomab, the company’s immunotherapy treatment for acute lymphoblastic leukemia (ALL), an aggressive blood cancer. The BLA application, and a Marketing Authorization Application (MAA) submitted to the European Medicines Agency (EMA), were based on the results of a Phase II study of blinatumomab, a bispecific T cell engager (BiTE) antibody construct, which met its primary endpoint for adult patients with a specific type of ALL, called Ph- relapsed/refractory B-precursor ALL.
“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,” Sean Harper, executive vice president of Research and Development at Amgen was quoted as saying in the press release. “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.”
Amgen’s BiTE antibody constructs, of which blinatumomab is the first example, aid the body’s immune system in detecting and targeting cancer cells. BiTE therapy places T cells, white blood cells that can kill other cells, within reach of a target cell so that the T cells can inject toxins into the cancer cell to trigger cell death (apoptosis). Blinatumomab has received orphan drug designation from the EMA and the FDA, and breakthrough therapy and priority review designation from the FDA for the treatment of ALL.
According to the FDA, “the Biologics License Application (BLA) is a request for permission to introduce, or deliver for introduction, a biologic product into interstate commerce.” The “priority review designation” indicates that the agency will take action on the application within six months of the application’s receipt. This designation is reserved for drugs that both treat serious conditions and represent a significant improvement over existing treatments.
More than 6,000 new cases of ALL will be diagnosed this year in the U.S. and more than 7,000 in the European Union. The estimated overall survival time for patients diagnosed with relapsed or refractory ALL is three to five months, indicating the pressing need for new more effective treatments for these patients.
