SAN DIEGO--(BUSINESS WIRE)--
Retrophin, Inc. (NASDAQ:RTRX) today announced the U.S. Food and Drug
Administration (FDA) has granted Fast Track designation for RE-024, the
Company's novel investigational phosphopantothenate replacement therapy,
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 is currently enrolling healthy volunteers in its Phase 1
clinical trial evaluating the safety and tolerability of single oral
doses of RE-024.
Fast Track designation is intended to aid the development of drugs to
treat serious conditions with unmet medical needs, and expedite the
review process with a goal of reaching patients earlier. A drug that
receives Fast Track designation may be eligible for more frequent
communication with the FDA to discuss development plans and clinical
trial design, as well as Accelerated Approval and Priority Review. The
developing company may also be eligible for Rolling Review, which
enables the submission of completed sections of its New Drug Application
(NDA) for review by the FDA before the company submits a complete
application.
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.
View source version on businesswire.com: http://www.businesswire.com/news/home/20150604006436/en/
Retrophin, Inc.
Chris Cline, CFA, 646-564-3680
Manager,
Investor Relations
IR@retrophin.com
Source: Retrophin, Inc.
News Provided by Acquire Media