Amgen Inc. (AMGN) Tuesday said the U.S. Food and Drug Administration has granted Breakthrough Therapy Designation to investigational bispecific T cell engager or BiTE antibody blinatumomab, for adults with Philadelphia-negative relapsed/refractory B-precursor acute lymphoblastic leukemia or ALL, a rapidly progressing cancer of the blood and bone marrow.
The Breakthrough Therapy Designation was based on the results of a Phase 2 trial of 189 adult patients with Ph- relapsed/refractory B-precursor ALL treated with blinatumomab.
Data from the Phase 2 trial were most recently presented at the 50th Annual Meeting of the American Society of Clinical Oncology and the 19th Congress of the European Hematology Association.
Breakthrough Therapy Designation is intended to expedite the development and review of drugs for serious or life-threatening conditions. The criteria for this designation require preliminary clinical evidence that demonstrates the drug may have substantial improvement on at least one clinically significant endpoint over available therapy.
A Breakthrough Therapy Designation conveys all of the fast-track program features, more intensive FDA guidance on an efficient drug development program, an organizational commitment involving senior managers, and eligibility for rolling review and priority review.
In the U.S. alone, it is estimated that over 6,000 cases of ALL were diagnosed in 2013. In adult patients with relapsed or refractory ALL, median overall survival is just three to five months.
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