Retrophin, Inc. (NASDAQ:RTRX) today announced that it has made RE-024
available to physicians who are treating critically ill patients with
pantothenate kinase-associated neurodegeneration (PKAN) worldwide under
local “compassionate use” regulations. RE-024 is a replacement therapy
for phosphopantothenate, the reaction product and substrate that is
missing in patients with PKAN.
A European health regulator approved the initiation of dosing RE-024 in
PKAN under a named patient program late last week. These patients will
be the first humans to receive RE-024. Retrophin expects that up to 15
patients will receive the drug as their physicians receive permission to
dose in countries across the world. Retrophin is working closely with
the U.S. Food and Drug Administration to enable as rapid access to
RE-024 in the United States as possible.
Retrophin’s Founder and Chief Executive Officer Martin Shkreli noted,
“PKAN is a deadly disease that has never received attention from
pharmaceutical companies. We are proud to be part of this important step
forward for patients and their families. We are eager to learn how
patients taking RE-024 fare as we move towards a placebo-controlled
double-blind study.”
About PKAN
PKAN is a rare and life-threatening neurological disorder caused by a
mutation in the PANK2 gene, which prevents patients from being able to
properly metabolize vitamin B5 (pantothenate) into phosphopantothenate.
The disruption of this metabolic pathway ultimately leads to decreased
levels of Coenzyme A (CoA) and iron accumulation in the brain. As a
result, patients present with dystonia (sustained muscle contraction
leading to abnormal posture), rigidity, dysphagia (problems swallowing),
weakness, pigmentary retinopathy (visual impairment), tremors, as well
as a number of other symptoms. Onset of PKAN typically occurs prior to
the age of 10 and has an estimated prevalence of 5,000-10,000 patients
worldwide. Many patients die from PKAN within 10 years of being
diagnosed.
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 marketed products include Chenodal®
and Vecamyl®, and its pipeline includes compounds for several
catastrophic diseases, including focal segmental glomerulosclerosis
(FSGS), pantothenate kinase-associated neurodegeneration (PKAN),
schizophrenia, autism, infantile spasms, nephrotic syndrome and others.
Retrophin intends to reintroduce Syntocinon Nasal Spray in the U.S. to
assist initial postpartum milk ejection. 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. Forward-looking statements in the press release should be
evaluated together with the many uncertainties that affect the Company's
business. 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 any 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.
Copyright Business Wire 2014