La Jolla Pharmaceutical Company Receives Orphan Drug Designation for Two Novel Compounds for Fibrodysplasia Ossificans Progre...
August 18 2015 - 8:05AM
Business Wire
La Jolla Pharmaceutical Company (Nasdaq: LJPC) (the Company or
La Jolla), a leader in the development of innovative therapies
intended to significantly improve outcomes in patients suffering
from life-threatening diseases, today announced that the U.S. Food
and Drug Administration (FDA) Office of Orphan Products Development
has granted La Jolla orphan drug designation for two novel
compounds for fibrodysplasia ossificans progressiva (FOP).
The compounds that received orphan drug designation are
small-molecule kinase inhibitors designed to selectively block a
specific member of the bone morphogenetic protein (BMP) type-I
receptor family, ALK2. La Jolla recently entered a worldwide,
exclusive license agreement with Vanderbilt University covering
this technology. The seven members of the BMP type-I receptor
family, activin receptor-like kinase (ALK) 1-7, play critical roles
in human development and physiology. In turn, the improper
activation of these receptor pathways is responsible for a wide
range of disease conditions.
FOP is a rare genetic disorder where the body turns muscle into
bone. FOP is caused by a genetic mutation in ALK2 that results in
excessive signaling of this pathway. In early childhood, afflicted
individuals develop soft tissue swellings that transform into bone.
Development of such lesions is exacerbated by trauma, and surgical
intervention leads to dramatic and explosive new bone growth. The
median survival of FOP patients is approximately 40 years, and
death often results from complications of thoracic insufficiency
syndrome, which is the inability of the thorax to support normal
respiration.
“There are no known treatment options available for patients
suffering from FOP, and the orphan drug designations recognize the
significant unmet need that exists within this disease,” said
George F. Tidmarsh, M.D., Ph.D., President and Chief Executive
Officer of La Jolla. “We look forward to further collaborating with
our colleagues at Vanderbilt University to find a potential
treatment for these patients in need.”
About Orphan Drug Designation
Orphan drug designation is granted by the FDA Office of Orphan
Products Development to novel drugs or biologics that treat a rare
disease or condition affecting fewer than 200,000 patients in the
U.S. The designation provides the drug developer with a seven-year
period of U.S. marketing exclusivity if the drug is the first of
its type approved for the specified indication or if it
demonstrates superior safety, efficacy, or a major contribution to
patient care versus another drug of its type previously granted the
designation for the same indication. The designation also provides
the drug developer with tax credits for clinical research costs,
the ability to apply for annual grant funding, clinical research
trial design assistance and a waiver of Prescription Drug User Fee
Act (PDUFA) filing fees.
About La Jolla Pharmaceutical Company
La Jolla Pharmaceutical Company is a biopharmaceutical company
focused on the discovery, development and commercialization of
innovative therapies intended to significantly improve outcomes in
patients suffering from life-threatening diseases. The Company has
several product candidates in development. LJPC-501 is La Jolla’s
proprietary formulation of angiotensin II for the potential
treatment of catecholamine-resistant hypotension. LJPC-401 is La
Jolla’s novel formulation of hepcidin for the potential treatment
of iron overload, which occurs as a result of diseases such as
hereditary hemochromatosis and beta thalassemia. LJPC-30Sa and
LJPC-30Sb are La Jolla’s next-generation gentamicin derivatives for
the potential treatment of serious bacterial infections and rare
genetic disorders, such as cystic fibrosis and Duchenne muscular
dystrophy. For more information on La Jolla, please visit
www.ljpc.com.
Forward-Looking Statement Safe Harbor
This document contains forward-looking statements as that term
is defined in the Private Securities Litigation Reform Act of 1995.
These statements relate to future events or the Company’s future
results of operations. These statements are only predictions and
involve known and unknown risks, uncertainties and other factors,
which may cause actual results to be materially different from
these forward-looking statements. The Company cautions readers not
to place undue reliance on any such forward-looking statements,
which speak only as of the date they were made. Certain of these
risks, uncertainties, and other factors are described in greater
detail in the Company’s filings with the U.S. Securities and
Exchange Commission (SEC), all of which are available free of
charge on the SEC’s web site www.sec.gov. These risks include, but
are not limited to, risks relating to: the timing for the filing of
an Investigational New Drug Application (IND), commencement of
clinical studies and the anticipated timing for completion of such
studies; the success of future development activities for the
Company’s drug candidates; potential indications for which the
Company’s drug candidates may be developed; and the Company’s
ability to obtain the potential benefits of orphan drug designation
for its drug candidates. Subsequent written and oral
forward-looking statements attributable to the Company or to
persons acting on its behalf are expressly qualified in their
entirety by the cautionary statements set forth in the Company’s
reports filed with the SEC. The Company expressly disclaims any
intent to update any forward-looking statements.
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version on businesswire.com: http://www.businesswire.com/news/home/20150818005500/en/
La Jolla Pharmaceutical CompanyGeorge F. Tidmarsh, M.D.,
Ph.D.President & Chief Executive
Officer858-207-4264gtidmarsh@ljpc.comorDennis M. MulroyChief
Financial Officer858-433-6839dmulroy@ljpc.com
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