Ligand Partner GlaxoSmithKline Gains Priority Review Designation for Promacta™/Revolade™ for Severe Aplastic Anemia
SAN DIEGO--
Ligand Pharmaceuticals Incorporated (NASDAQ: LGND) today
announced that the U.S. Food and Drug Administration (FDA) has granted
its partner GlaxoSmithKline plc (LSE:GSK) 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
About Promacta
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 visit www.captisol.com
for more information on Captisol and www.ligand.com
for more information on Ligand.
Follow Ligand on Twitter @Ligand_LGND.
References
i Valdez JM, et al. Decreased Infection-Related Mortality and
Improved Survival in Severe Aplastic Anemia in the Past Two Decades.
Clinical Infectious Diseases 2011;52(6):726–735.
ii U.S. Food and Drug Administration. Fast Track,
Breakthrough Therapy, Accelerated Approval and Priority Review.
Available at: http://www.fda.gov/forconsumers/byaudience/forpatientadvocates/speedingaccesstoimportantnewtherapies/ucm128291.htm.
Accessed April 28, 2014.
Forward-Looking Statements
This news release contains forward-looking statements by Ligand that
involve risks and uncertainties and reflect Ligand’s judgment as of the
date of this release. These forward-looking statements include comments
regarding eltrombopag and other drug candidates, data analysis and
evaluation of eltrombopag, utility or potential benefits to patients,
the potential commercial market for eltrombopag and plans for continued
development and further studies of eltrombopag. Actual events or results
may differ from Ligand’s expectations. For example, there can be no
assurance that other trials or evaluations of eltrombopag or other
product candidates will be favorable or that they will confirm results
of previous studies, that data evaluation will be completed or
demonstrate any hypothesis or endpoint, that eltrombopag or other
product candidates will provide utility or benefits to certain patients,
that any presentations will be favorably received, that eltrombopag or
other product candidates will be useful, that marketing applications
will be filed or, if filed, approved, or that clinical or commercial
development of these product candidates will be initiated, completed or
successful or that our rights to eltrombopag and other related product
candidates will not be successfully challenged. The failure to meet
expectations with respect to any of the foregoing matters may reduce
Ligand’s stock price. Additional information concerning these and other
risk factors affecting Ligand can be found in prior press releases
available at www.ligand.com
as well as in public periodic filings with the Securities and Exchange
Commission, available at www.sec.gov.
Ligand disclaims any intent or obligation to update these
forward-looking statements beyond the date of this press release. This
caution is made under the safe harbor provisions of the Private
Securities Litigation Reform Act of 1995.
Ligand Pharmaceuticals Incorporated
John L. Higgins, President and
CEO
858-550-7500
or
LHA
Bruce Voss, 310-691-7100
bvoss@lhai.com
Source: Ligand Pharmaceuticals Incorporated
Released April 30, 2014