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DGAP-News: Raptor Pharmaceutical Corp. Submits New Drug Application for RP103 for the Potential Treatment of Nephropathic Cystinosis
ID: 608165
(firmenpresse) - Raptor Pharmaceutical Corp.
30.03.2012 17:44
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NOVATO, Calif., 2012-03-30 17:44 CEST (GLOBE NEWSWIRE) --
Raptor Pharmaceutical Corp. ('Raptor' or the 'Company') (Nasdaq:RPTP) announced
that it submitted a New Drug Application ('NDA') to the U.S. Food and Drug
Administration ('FDA') seeking approval to market its investigational drug
candidate, Cysteamine Bitartrate Delayed-release Capsules (RP103), for the
potential treatment of nephropathic cystinosis. In its application, Raptor has
requested Priority Review of the NDA, which, if granted, could lead to a
decision for marketing approval from the FDA of RP103 for the potential
treatment of nephropathic cystinosis in the fourth calendar quarter of 2012.
Earlier this week, on March 26, Raptor announced European Medicines Agency
('EMA') validation of its Marketing Authorization Application ('MAA') for RP103
for the potential treatment of nephropathic cystinosis.
'A Company's first NDA submission is a significant milestone and we are proud
of everyone who helped us to achieve this goal,' said Christopher M. Starr,
Ph.D., Raptor's Chief Executive Officer. 'We could not be more pleased to have
now submitted both our NDA, as well as our MAA to the EMA, of RP103 for the
potential treatment of cystinosis, by taking advantage of the newly recommended
electronic Common Technical Document ('eCTD') format.'
The NDA submission for marketing approval includes previously announced data
from Raptor's Phase 3 clinical trial of RP103. As reported, RP103 met the
clinical trial's sole primary endpoint and there were no unexpected serious
safety concerns attributable to RP103 experienced by patients in the trial. The
trial was conducted at three clinical sites in the U.S. and five clinical sites
in Europe.
Patrice P. Rioux, M.D., Ph.D., Raptor's Chief Medical Officer, said, 'We
believe the positive clinical data generated by our Phase 3 clinical trial
demonstrates that RP103 is safe and effective in cystinosis patients. We look
forward to working closely with FDA in their review of our application.'
About Nephropathic Cystinosis
Nephropathic cystinosis, an orphan disease, is estimated to effect a population
of 2,000 patients worldwide, including 500 patients in the U.S. and 800
patients in Europe. Cystinosis patients have inherited a defective cystine
transporter gene which results in body-wide cellular toxicity resulting from
the abnormal buildup of the amino acid cystine in the lysosomes. Cystinosis is
usually diagnosed in the first year of life and requires lifelong therapy.
Cystine crystals accumulate in various tissues and organs, including the
kidneys, brain, liver, thyroid, pancreas, muscles and eyes. Left untreated, the
disease is fatal by the first decade of life. RP103 reduces cellular toxicity
by continuously removing cystine from the lysosome.
About Cysteamine and RP103
RP103 is Raptor's proprietary delayed and extended release oral medication
designed to treat the underlying metabolic cause of cystinosis. RP103 is an
enteric coated, microbead formulation of cysteamine bitartrate that has been
formulated to be sprinkled onto food for administration to patients too young
to take oral capsules. 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 of RP103 for
nephropathic cystinosis and for cysteamine for other potential indications
including Huntington's Disease currently in a Phase 2/3 clinical trial in
France and non-alcoholic steatohepatitis ('NASH') currently in a Phase 2b
clinical trial in the U.S.
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
http://www.globenewswire.com/newsroom/prs/?pkgid=7180
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 RP103
will receive Priority Review and marketing approval from the FDA of RP103 for
the treatment of nephropathic cystinosis in the fourth calendar quarter of
2012, if at all; that RP103 is safe and effective in cystinosis patients;
that the FDA will expedite their review of Raptor's application; that RP103 for
the treatment of nephropathic cystinosis will be approved by the EMA; that
Raptor will successfully commercialize RP103, if approved; and that Raptor will
be able to successfully develop 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 and Amendment No. 1 to its Annual Report on
Form 10-K/A filed with the SEC on November 14, 2011 and December 19, 2011,
respectively; and Raptor's quarterly report on Form 10-Q filed with the SEC on
January 6, 2012, which are available free of charge on the SEC's web site athttp://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)
Lauren Glaser
(646) 378-2972
lglaser(at)troutgroup.com
EVC Group (media)
Janine McCargo
(646) 688-0425
jmccargo(at)evcgroup.com
News Source: NASDAQ OMX
30.03.2012 Dissemination of a Corporate News, transmitted by DGAP -
a company of EquityStory AG.
The issuer is solely responsible for the content of this announcement.
DGAP's Distribution Services include Regulatory Announcements,
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Media archive at www.dgap-medientreff.de and www.dgap.de
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Language: English
Company: Raptor Pharmaceutical Corp.
United States
Phone:
Fax:
E-mail:
Internet:
ISIN: US75382F1066
WKN:
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Datum: 30.03.2012 - 17:44 Uhr
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