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FDA Breakthrough Therapy Designation: Amgen Gets #45

July 1, 2014

Amgen receives on July 1st, the FDA Breakthrough Therapy Designation (BTD) for the investigational bispecific T cell engager (BiTE) antibody Blinatumomab. The BTD is for the treatment of adults with Philadelphia-negative (Ph-) relapsed/refractory B-precursor Acute Lymphoblastic Leukemia (ALL). ALL is an aggressive cancer of the blood and bone marrow that progresses rapidly. Patients with ALL have abnormal white blood cells that “crowd out healthy white blood cells, red blood cells and platelets, leading to infection, anemia, easy bleeding and serious side effects”.

This is the 45th BTD announced by a sponsor company and the 1st BTD for Amgen. Blinatumomab has FDA Orphan Drug Designations (ODDs) for the following 5 oncology indications:

Num FDA ODD Date Indication
1 05.16.2008 Chronic Lymphocytic Leukemia
2 02.06.2006 Indolent B-cell Lymphoma, excluding CLL and NHL with CNS involvement
3 05.16.2008 Acute Lymphocytic Leukemia
4 05.16.2008 Prolymphocytic Leukemia
5 05.16.2008 Hairy Cell Leukemia


ALL Statistics

•   Accounts for > 12% of Leukemia

•   42,000 are diagnosed worldwide with 31,000 or approximately 74% dying of the disease

•   In US, 6,000 cases diagnosed in 2013

•   In EU, > 7,000 cases diagnosed yearly

•   Median overall survival is just 3 – 5 months for Relapsed/Refractory ALL.



FDA BTD Approval Chart

FDA BTD Statistics Chart.

Please Note: “Paraglider ridge soaring at Torrey Pines”  by Pastelitodepapa (Own work) [GFDL or CC-BY-SA-3.0], via Wikimedia Commons.

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