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FDA Breakthrough Therapy Designation: Novartis Gets A 5th

July 8, 2014

Novartis receives on July 7th, the FDA Breakthrough Therapy Designation (BTD) for CTL09. The BTD is for the treatment of pediatric and adult patients with Relapsed/Refractory Acute Lymphoblastic Leukemia (R/R ALL). CTL09 is an:

“ … investigational Chimeric Antigen Receptor (CAR) therapy … (that) uses technology to reprogram a patient’s own T cells to “hunt” cancer cells that express specific proteins, called CD19. After they have been reprogrammed, the T cells are re-introduced into the patient’s blood; they proliferate and bind to the targeted CD19+ cancer cells and destroy them.”

The University of Pennsylvania’s Perelman School of Medicine (UPPSM) filed for the BTD. UPPSM and Novartis have a global agreement to research, develop, and commercialize personalized CAR T Cell Therapies for cancers.

This is the 46th BTD announced by a sponsor company and the 5th BTD for Novartis :

Num Drug Name Indication
1 LDK378 (Zykadia) ** ALK+


Cell Lung

Cancer (NSCLC)

2 Serelaxin Acute Heart


3 Bimagrumab Sporadic


Body Myositis

4 Bexsero Vaccine Meningococcal BDisease
5 Personalized Cell Therapy CTL019 Relapsed/refractory acute lymphoblastic leukemia (r/r ALL)


** Zykadia receives FDA Approval in April 2014.

Just last week, Amgen receives the FDA BTD for the investigational bispecific T cell engager (BiTE) antibody Blinatumomab, for the treatment of adults with Philadelphia-negative (Ph-) relapsed/refractory B-precursor Acute Lymphoblastic Leukemia (ALL).



FDA BTD Approval Chart

FDA BTD Statistics Chart.

Please Note: “Glowing heart (114668824)” by 29cm from Hong Kong (Glowing heart) [CC-BY-SA-2.0] | Wikimedia Commons.

Copyright © 2012-2014, Orphan Druganaut Blog. All rights reserved.

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