GSK gains FDA Breakthrough Therapy designation for Promacta®/Revolade® (eltrombopag) for severe aplastic anaemia
Issued: London UK
GlaxoSmithKline plc (LSE:GSK) announced today that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for Promacta®/Revolade® (eltrombopag) for the treatment of cytopenias in patients with severe aplastic anaemia (SAA) who have had insufficient response to immunosuppressive therapy. Eltrombopag is not approved or licensed anywhere in the world for use in this treatment setting. Breakthrough Therapy Designation is the newest of the FDA’s programmes aimed at accelerating the development and review times of drugs for serious or life-threatening conditions.i
SAA is a rare disorder in which the bone marrow fails to make enough new blood cells. There are no approved therapies available for SAA patients unresponsive to initial immunosuppressive therapy (IST). Of those patients unresponsive to initial IST, approximately 40% die from infection or bleeding within 5 years of their diagnosis.ii
The Breakthrough Therapy designation was based on the results from an open-label, Phase II National Institute of Health (NIH) study (09-H-0154) of eltrombopag in 43 heavily pre-treated SAA patients with an insufficient response to IST.
About Breakthrough Therapy Designation
The Breakthrough Therapy designation was enacted as part of the 2012 FDA Safety and Innovation Act (FDASIA) and is intended to expedite development and review of drugs to treat serious or life-threatening medical conditions when preliminary clinical evidence demonstrates that the drug may have substantial improvement on at least one clinically significant endpoint over available therapies. Breakthrough Therapy designation includes all the features of the Fast Track designation, as well as more intensive guidance from the FDA on a drug’s clinical development programme.i
For full U.S. Prescribing Information and Medication Guide and EU Patient Information Leaflet or Summary of Product Characteristics (SPC) for eltrombopag please visit http://www.gsk.com/products/
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Cautionary statement regarding forward-looking statements
GSK cautions investors that any forward-looking statements or projections made by GSK, including those made in this announcement, are subject to risks and uncertainties that may cause actual results to differ materially from those projected. Factors that may affect GSK' s operations are described under Item 3.D 'Risk factors' in the company's Annual Report on Form 20-F for 2012.