SAN DIEGO--(BUSINESS WIRE)--
Retrophin, Inc. (NASDAQ:RTRX) today announced RE-024, the Company's
investigational phosphopantothenate replacement therapy, has been
granted orphan drug designation by the U.S. Food and Drug Administration
(FDA) for the treatment of pantothenate kinase-associated
neurodegeneration (PKAN). PKAN is a rare and lethal autosomal recessive
neurodegenerative disorder believed to affect approximately one to three
persons per million worldwide, with no approved treatment currently
available. Retrophin has initiated the screening of healthy volunteers
for its recently announced Phase 1 trial evaluating the safety and
tolerability of single oral doses of RE-024.
The Orphan Drug Designation program is intended to encourage companies
to develop therapeutics for diseases that affect fewer than 200,000
individuals in the United States. Orphan designation will provide RE-024
with seven years of marketing exclusivity if approved by the FDA for the
treatment of PKAN. Prior to FDA approval, orphan designation provides
incentives for sponsors including tax credits for clinical research
expenses, the opportunity to obtain government grant funding to support
clinical research, and an exemption from FDA user fees.
About Retrophin
Retrophin is a pharmaceutical company focused on the development,
acquisition and commercialization of drugs for the treatment of serious,
catastrophic or rare diseases for which there are currently no viable
options for patients. The Company's approved products include Chenodal®,
Cholbam™, and Thiola®, and its pipeline includes compounds for several
catastrophic diseases, including focal segmental glomerulosclerosis
(FSGS), pantothenate kinase-associated neurodegeneration (PKAN),
infantile spasms, nephrotic syndrome and others. For additional
information, please visit www.retrophin.com.
Forward-Looking Statements
This press release contains "forward-looking statements" as that term is
defined in the Private Securities Litigation Reform Act of 1995,
regarding the research, development and commercialization of
pharmaceutical products. Without limiting the foregoing, these
statements are often identified by the words "may", "might", "believes",
"thinks", "anticipates", "plans", "expects", "intends" or similar
expressions. In addition, expressions of our strategies, intentions or
plans are also forward-looking statements. Such forward-looking
statements are based on current expectations and involve inherent risks
and uncertainties, including factors that could delay, divert or change
any of them, and could cause actual outcomes and results to differ
materially from current expectations. No forward-looking statement can
be guaranteed. Among the factors that could cause actual results to
differ materially from those indicated in the forward-looking statements
are risks and uncertainties associated with the Company's business and
finances in general, as well as risks and uncertainties associated with
the Company's pre-clinical and clinical stage pipeline as well as its
sales and marketing strategies. Specifically, the risks and
uncertainties the Company faces with respect to its RE-024 program
include risk that the Company will be unable to initiate and/or complete
Phase 1 clinical trials, risk that RE-024 will not progress to Phase 2
clinical trials for safety, regulatory or other reasons; risk associated
with enrollment of clinical trials for rare diseases; risk that the
company's later stage RE-024 clinical studies will fail to demonstrate
that RE-024 is safe and effective. The Company also faces risk that it
will be unable to raise additional funding required to complete
development of any or all of its product candidates; risk relating to
the Company's dependence on contractors for clinical drug supply and
commercial manufacturing; uncertainties relating to patent protection
and intellectual property rights of third parties; risks and
uncertainties relating to competitive products and technological changes
that may limit demand for the Company's products. You are cautioned not
to place undue reliance on these forward-looking statements as there are
important factors that could cause actual results to differ materially
from those in forward-looking statements, many of which are beyond our
control. The Company undertakes no obligation to publicly update
forward-looking statement, whether as a result of new information,
future events, or otherwise. Investors are referred to the full
discussion of risks and uncertainties as included in the Company's
filings with the Securities and Exchange Commission.
Retrophin, Inc.
Chris Cline, CFA, 646-564-3680
Manager,
Investor Relations
IR@retrophin.com
Source: Retrophin, Inc.
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