AB Science: Masitinib Receives Orphan Drug Designation for Amyotrophic Lateral Sclerosis from FDA
March 20 2015 - 1:32PM
AB Science SA (NYSE Euronext - FR0010557264 - AB), a
pharmaceutical company specialized in research, development and
marketing of protein kinase inhibitors (PKIs), announces that the
U.S. Food and Drug Administration (FDA) has granted the company
Orphan Drug designation for masitinib in the treatment of
amyotrophic lateral sclerosis.
The FDA's Office of Orphan Drug Products
Development reviews applications for Orphan Drug status to support
development of medicines for underserved patient populations, or
rare disorders that affect fewer than 200,000 people in the United
States. The successful application submitted by AB Science and the
FDA granting of Orphan Drug status entitles the company to a
seven-year period of marketing exclusivity in the United States for
masitinib, if it is approved by the FDA for the treatment of
amyotrophic lateral sclerosis. Orphan Drug status also enables the
company to apply for research grant funding for Phase I and II
Clinical Trials, tax credits for certain research expenses, and a
waiver from the FDA's application user fee, as well as additional
support from FDA and a potentially faster regulatory process.
A phase 3 is currently on-going with masitinib
in ALS. In January 2015, the external Data and Safety Monitoring
Board (DSMB) recommended the continuation of this phase 3 study
based upon review of the latest safety data. The DSMB was created
as part of the Company's pivotal clinical study evaluating
masitinib in the treatment of amyotrophic lateral sclerosis.
Amyotrophic lateral sclerosis is a rare
degenerative disorder that results in progressive wasting and
paralysis of voluntary muscles. There are approximately 30,000
people with ALS in the European Union and 15,000 in the US, with
more than 7,500 new cases diagnosed each year in Europe and 4,500
in the US. Almost 50% of ALS patients die within 3 years and 90%
die within 5 years.
About Orphan Drug Designation
The FDA Office of Orphan Products Development
(OOPD) mission is to advance the evaluation and development of
products (drugs, biologics, devices, or medical foods) that
demonstrate promise for the diagnosis and/or treatment of rare
diseases or conditions. In fulfilling that task, OOPD evaluates
scientific and clinical data submissions from sponsors to identify
and designate products as promising for rare diseases and to
further advance scientific development of such promising medical
products.
The approval of an orphan designation request
does not alter the standard regulatory requirements and process for
obtaining marketing approval for investigational use. Sponsors must
establish safety and efficacy of a compound in the treatment of a
disease through adequate and well-controlled studies. However, the
FDA review process may be speedier for Orphan Drugs than those
which do not receive Orphan Drug designation.
About masitinib
Masitinib is a new orally administered tyrosine
kinase inhibitor that targets mast cells and macrophages, important
cells for immunity, through inhibiting a limited number of kinases.
Based on its unique mechanism of action, masitinib can be developed
in a large number of conditions in oncology, in inflammatory
diseases, and in certain diseases of the central nervous system. In
oncology due to its immunotherapy effect, masitinib can have an
effect on survival, alone or in combination with chemotherapy.
Through its activity on mast cells and consequently the inhibition
of the activation of the inflammatory process, masitinib can have
an effect on the symptoms associated with some inflammatory and
central nervous system diseases and the degeneration of these
diseases.
About AB Science
Founded in 2001, AB Science is a pharmaceutical
company specializing in the research, development and
commercialization of protein kinase inhibitors (PKIs), a
class of targeted proteins whose action are key in signaling
pathways within cells. Our programs target only diseases with high
unmet medical needs, often lethal with short term survival or rare
or refractory to previous line of treatment in cancers,
inflammatory diseases, and central nervous system diseases, both in
humans and animal health.
AB Science has developed a proprietary portfolio
of molecules and the Company's lead compound, masitinib, has
already been registered for veterinary medicine in Europe and in
the USA. The company is currently pursuing thirteen phase 3 studies
in human medicine in first-line and second-line GIST, metastatic
melanoma expressing JM mutation of c-Kit, multiple myeloma,
metastatic colorectal cancer, metastatic prostate cancer,
pancreatic cancer, mastocytosis, severe persistent asthma,
rheumatoid arthritis, Alzheimer's disease, progressive forms of
multiple sclerosis, and Amyotrophic Lateral Sclerosis. The company
is headquartered in Paris, France, and listed on Euronext Paris
(ticker: AB).
Further information is available on AB Science
website: www.ab-science.com.
This document contains prospective information.
No guarantee can be given as for the realization of these
forecasts, which are subject to those risks described in documents
deposited by the Company to the Authority of the financial markets,
including trends of the economic conjuncture, the financial markets
and the markets on which AB Science is present.
AB Science - Financial Communication & Media
Relations investors@ab-science.com
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