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FDA Breakthrough Therapy Designation: 32 And Counting

February 3, 2014

On February 3rd, GlaxoSmithKline (GSK) announces that Promacta (US)/Revolade (Europe) (Eltrombopag) receives the coveted FDA Breakthrough Therapy Designation (BTD) for cytopenias in patients with Severe Aplastic Anemia (SAA), who have had insufficient response to Immunosuppressive Therapy (IST). The drug is not approved or licensed anywhere in the world for this indication.

SAA is a rare disorder where the bone marrow fails to make enough new blood cells. There are currently no therapies approved for this indication. About forty percent (40%) of patients who do not respond to initial IST die within 5 years of diagnosis.

Regulatory Actions

•   Receives FDA ODD in November 2013 for Aplastic Anemia

•   Receives FDA BTD in February 2014 for Aplastic Anemia

•   Receives FDA ODD in May 2008 & FDA approval in November 2008 for Idiopathic Thrombocytopenia Purpura.

This is the 32nd BTD that is announced by a sponsor company since FDASIA is signed into law in July 2012. It is the 2nd BTD so far for 2014. This is the 5th FDA BTD that GSK receives – the most by any sponsor company identified so far:

Row Num

Drug Name

Sponsor                         Company Indication


Drisapersen GSK Duchenne     MuscularDystrophy


Ofatumumab Genmab/GSK Chronic     LymphocyticLeukemia (CLL)


Tafenoquine GSK/Medicines for Malaria     Venture Plasmodium Vivax       Malaria


Dabrafenib GSK Metastatic   BRAF V600E Mutation-Positive Non-Small Cell Lung Cancer   (NSCLC)


Eltrombopag GSK Severe   Aplastic Anemia


The Column in the Chart titled “Drug Name” contains the URL for the link to the Press Release announcing the FDA Breakthrough Therapy Designation for each drug.

Please Note: FDA Official Logo from FDA website.

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

  1. All for One permalink

    Do you know what company was granted BTS for CBER.

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