Raptor Pharmaceutical Corp. Holds Pre-NDA Meeting With FDA for RP103 for Nephropathic Cystinosis
Raptor Pharmaceutical Corp.
Raptor Pharmaceutical Corp. Holds Pre-NDA Meeting With FDA for RP103 for
Company Remains on Track for NDA Submission in Q1 2012
NOVATO, Calif., 2011-10-25 20:27 CEST (GLOBE NEWSWIRE) -- Raptor Pharmaceutical
Corp. ("Raptor" or the "Company") (Nasdaq:RPTP), announced today that the
Company concluded a pre-New Drug Application ("NDA") meeting with the U.S. Food
and Drug Administration ("FDA") related to its investigational drug candidate,
RP103 (delayed-release formulation of cysteamine bitartrate), for the treatment
of nephropathic cystinosis. The purpose of the meeting was to discuss the
proposed NDA and to confirm the clinical, non-clinical and manufacturing
requirements for the Company's NDA submission, which the Company intends to
file in the first quarter of 2012, as previously announced.
"We've come out of the pre-NDA meeting with a bolstered understanding of the
agency's requirements for our submission," said Christopher M. Starr, Ph.D. and
CEO of Raptor. "Based on feedback received during the meeting and in prior
correspondence, we are moving forward with our NDA submission for RP103 in the
first quarter of next year."
About Nephropathic Cystinosis
Nephropathic cystinosis is an inborn metabolic error characterized by the
abnormal transport of cystine, an amino acid, out of the lysosomes. Poor
compliance with current treatments for nephropathic cystinosis can cause
serious health consequences, including: renal failure and resultant need for a
kidney transplant; growth failure; rickets and fractures; and photophobia and
blindness. Symptom onset typically occurs within the first year of life, when
cystine crystals accumulate in various tissues and organs, including the
kidneys, brain, liver, thyroid, pancreas, muscles and eyes.
About Cysteamine and RP103
RP103 is Raptor's proprietary enteric-coated, microbead oral formulation of
cysteamine bitartrate designed to potentially reduce dosing frequency and
reduce gastrointestinal side effects associated with immediate-release
cysteamine bitartrate, which is approved for sale by the FDA and the European
Medicines Agency ("EMA") to treat nephropathic cystinosis. Raptor has been
granted orphan product designation for RP103 by the EMA and FDA.
In December 2007, Raptor obtained an exclusive, worldwide license from the
University of California, San Diego for the development DR Cysteamine for
nephropathic cystinosis and cysteamine for other potential indications
including Huntington's Disease and Non-alcoholic Steatohepatitis.
About Raptor Pharmaceutical Corp.
Raptor Pharmaceutical Corp. (Nasdaq:RPTP) ("Raptor") seeks to research,
produce, and deliver medicines that improve life for patients with severe, rare
disorders. Raptor currently has product candidates in clinical development
designed to potentially treat nephropathic cystinosis, Non-alcoholic
Steatohepatitis ("NASH"), Huntington's Disease ("HD"), aldehyde dehydrogenase
deficiency ("ALDH2"), and thrombotic disorder.
Raptor's preclinical programs are based upon bioengineered novel drug
candidates and drug-targeting platforms derived from the human
receptor-associated protein and related proteins that are designed to target
cancer and infectious diseases.
For additional information, please visit www.raptorpharma.com.
The Raptor Pharmaceutical Corp. logo is available at
FORWARD LOOKING STATEMENTS
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 our future results of operation or future financial
performance, including, but not limited to the following statements:; that
Raptor will file a New Drug Application in the first quarter of 2012, if at
all; and that Raptor will be able to successfully develop RP103 or DR
Cysteamine or any of its other product candidates. These statements are only
predictions and involve known and unknown risks, uncertainties and other
factors, which may cause the Company's actual results to be materially
different from these forward-looking statements. Factors which may
significantly change or prevent the Company's forward looking statements from
fruition include: that Raptor may be unsuccessful in developing any products or
acquiring products; that Raptor's technology may not be validated as it
progresses further and its methods may not be accepted by the scientific
community; that Raptor is unable to retain or attract key employees whose
knowledge is essential to the development of its products; that unforeseen
scientific difficulties develop with the Company's process; that Raptor's
patents are not sufficient to protect essential aspects of its technology; that
competitors may invent better technology; that Raptor's products may not work
as well as hoped or worse, that the Company's products may harm recipients; and
that Raptor may not be able to raise sufficient funds for development or
working capital. As well, Raptor's products may never develop into useful
products and even if they do, they may not be approved for sale to the public.
Raptor 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 from time to time with the Securities and Exchange Commission
(the "SEC"), which Raptor strongly urges you to read and consider, including:
Raptor's annual report on Form 10-K filed with the SEC on November 22, 2010;
and Raptor's quarterly report on Form 10-Q filed with the SEC on July 12, 2011;
all of which are available free of charge on the SEC's web site at
http://www.sec.gov. Subsequent written and oral forward-looking statements
attributable to Raptor or to persons acting on its behalf are expressly
qualified in their entirety by the cautionary statements set forth in Raptor's
reports filed with the SEC. Raptor expressly disclaims any intent or obligation
to update any forward-looking statements.
CONTACT: Trout Group (investors)
EVC Group (media)