Raptor Pharmaceutical Corp. Announces EMA Validation of Marketing Authorization Application for RP103 for the Treatment of Nephropathic Cystinosis
Raptor Pharmaceutical Corp.
26.03.2012 12:30
---------------------------------------------------------------------------
FDA NDA Submission Planned in March 2012
NOVATO, Calif., 2012-03-26 12:30 CEST (GLOBE NEWSWIRE) --
Raptor Pharmaceutical Corp. ('Raptor' or the 'Company') (Nasdaq:RPTP),
announced that the European Medicines Agency ('EMA') has determined that
Raptor's Marketing Authorization Application ('MAA') for its investigational
drug candidate, RP103 for the potential treatment of nephropathic cystinosis,
submitted earlier this month, is valid. Validation of the MAA confirms that the
submission is sufficiently complete to begin the formal review process.
'This filing represents the Company's first registration application and a
significant milestone in our journey to make RP103 available to cystinosis
patients throughout the world,' said Christopher M. Starr, Ph.D., Raptor's
Chief Executive Officer. 'As we move closer to potential approvals, we remain
dedicated to serving the cystinosis patients, medical community and foundations
and thank them for their critical roles in bringing us to this point in the
program.'
The MAA submission for registration includes data from Raptor's Phase 3
clinical trial of RP103 (cysteamine bitartrate gastro-resistant capsules),
which was announced in July 2011. 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.
'We are very appreciative of the rapid initial assessment and validation of our
application by EMA and look forward to working with the Committee for Medicinal
Products for Human Use ('CHMP') and our rapporteur and co-rapporteur on the MAA
review,' said Patrice P. Rioux, M.D., Ph.D., Raptor's Chief Medical Officer.
'In the meantime, we remain on track to file our NDA submission for RP103 for
the potential treatment of nephropathic cystinosis in the U.S. by the end of
March.'
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 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
Raptor will submit its NDA with the FDA by the end of March; that RP103 for the
treatment of nephropathic cystinosis will be approved by the EMA or FDA; 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 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)
Lauren Glaser
(646) 378-2972
lglaser@troutgroup.com
EVC Group (media)
Janine McCargo
(646) 688-0425
jmccargo@evcgroup.com
News Source: NASDAQ OMX
26.03.2012 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,
Financial/Corporate News and Press Releases.
Media archive at www.dgap-medientreff.de and www.dgap.de
---------------------------------------------------------------------------
Language: English
Company: Raptor Pharmaceutical Corp.
United States
Phone:
Fax:
E-mail:
Internet:
ISIN: US75382F1066
WKN:
End of Announcement DGAP News-Service
---------------------------------------------------------------------------
Raptor Pharmaceutical Corp.
26.03.2012 12:30
---------------------------------------------------------------------------
FDA NDA Submission Planned in March 2012
NOVATO, Calif., 2012-03-26 12:30 CEST (GLOBE NEWSWIRE) --
Raptor Pharmaceutical Corp. ('Raptor' or the 'Company') (Nasdaq:RPTP),
announced that the European Medicines Agency ('EMA') has determined that
Raptor's Marketing Authorization Application ('MAA') for its investigational
drug candidate, RP103 for the potential treatment of nephropathic cystinosis,
submitted earlier this month, is valid. Validation of the MAA confirms that the
submission is sufficiently complete to begin the formal review process.
'This filing represents the Company's first registration application and a
significant milestone in our journey to make RP103 available to cystinosis
patients throughout the world,' said Christopher M. Starr, Ph.D., Raptor's
Chief Executive Officer. 'As we move closer to potential approvals, we remain
dedicated to serving the cystinosis patients, medical community and foundations
and thank them for their critical roles in bringing us to this point in the
program.'
The MAA submission for registration includes data from Raptor's Phase 3
clinical trial of RP103 (cysteamine bitartrate gastro-resistant capsules),
which was announced in July 2011. 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.
'We are very appreciative of the rapid initial assessment and validation of our
application by EMA and look forward to working with the Committee for Medicinal
Products for Human Use ('CHMP') and our rapporteur and co-rapporteur on the MAA
review,' said Patrice P. Rioux, M.D., Ph.D., Raptor's Chief Medical Officer.
'In the meantime, we remain on track to file our NDA submission for RP103 for
the potential treatment of nephropathic cystinosis in the U.S. by the end of
March.'
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 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
Raptor will submit its NDA with the FDA by the end of March; that RP103 for the
treatment of nephropathic cystinosis will be approved by the EMA or FDA; 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 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)
Lauren Glaser
(646) 378-2972
lglaser@troutgroup.com
EVC Group (media)
Janine McCargo
(646) 688-0425
jmccargo@evcgroup.com
News Source: NASDAQ OMX
26.03.2012 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,
Financial/Corporate News and Press Releases.
Media archive at www.dgap-medientreff.de and www.dgap.de
---------------------------------------------------------------------------
Language: English
Company: Raptor Pharmaceutical Corp.
United States
Phone:
Fax:
E-mail:
Internet:
ISIN: US75382F1066
WKN:
End of Announcement DGAP News-Service
---------------------------------------------------------------------------