April 30, 2014
Ligand Pharmaceuticals Incorporated today announced that the U.S. Food and Drug Administration (FDA) has granted its partner GlaxoSmithKline plc Priority Review designation for Promacta™/Revolade™ (eltrombopag) for the treatment of cytopenias in patients with severe aplastic anemia (SAA) who have had insufficient response to immunosuppressive therapy.
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 who are unresponsive to initial immunosuppressive therapy (IST). Of those patients unresponsive to initial IST, approximately 40% die from infection or bleeding within five years of their diagnosis.i
In February 2014, Ligand announced that GSK had gained Breakthrough Therapy designation for Promacta in Severe Aplastic Anemia. Also in February 2014, GSK announced the submission of a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for Promacta for the treatment of cytopenias (a reduction in blood cells) in patients with SAA who have had an insufficient response to IST. The sNDA is 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 Priority Review Designation
A Priority Review designation is assigned to applications for drugs that, if approved, would provide significant improvements in the safety or effectiveness of the treatment, diagnosis or prevention of serious conditions and means FDA’s goal is to take action on an application within 6 months, compared to 10 months under standard review.
Significant improvement may be demonstrated by the following examples:
- Evidence of increased effectiveness in treatment, prevention, or diagnosis of condition;
- Elimination or substantial reduction of a treatment-limiting drug reaction;
- Documented enhancement of patient compliance that is expected to lead to an improvement in serious outcomes; or
- Evidence of safety and effectiveness in a new subpopulation.ii
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 program.ii
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.html.
Promacta is not approved or licensed anywhere in the world for use in SAA.
About Ligand Pharmaceuticals
Ligand is a biopharmaceutical company with a business model that is based upon the concept of developing or acquiring royalty revenue generating assets and coupling them to a lean corporate cost structure. Ligand’s goal is to produce a bottom line that supports a sustainably profitable business. By diversifying the portfolio of assets across numerous technology types, therapeutic areas, drug targets and industry partners, we offer investors an opportunity to invest in the increasingly complicated and unpredictable pharmaceutical industry. In comparison to its peers, we believe Ligand has assembled one of the largest and most diversified asset portfolios in the industry with the potential to generate revenue in the future. These therapies seek to address the unmet medical needs of patients for a broad spectrum of diseases including diabetes, hepatitis, muscle wasting, Alzheimer’s disease, dyslipidemia, anemia, asthma and osteoporosis. Ligand’s Captisol platform technology is a patent-protected, chemically modified cyclodextrin with a structure designed to optimize the solubility and stability of drugs. Ligand has established multiple alliances with the world’s leading pharmaceutical companies including GlaxoSmithKline, Onyx Pharmaceuticals (a subsidiary of Amgen Inc.), Merck, Pfizer, Baxter International, Lundbeck Inc., Eli Lilly & Co. and Spectrum Pharmaceuticals. Please visitwww.captisol.com for more information on Captisol and www.ligand.com for more information on Ligand.
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