DGAP-News: Raptor Pharmaceutical's PROCYSBI(TM) Receives Positive Recommendation From CHMP

ID: 1241380
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(businesspress24) - Raptor Pharmaceutical Corp.

28.06.2013 14:58
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NOVATO, Calif., 2013-06-28 14:58 CEST (GLOBE NEWSWIRE) --
Raptor Pharmaceutical Corp. (Nasdaq:RPTP) today announced today that the
European Committee for Medicinal Products for Human Use (CHMP) has adopted a
positive opinion recommending marketing authorization for PROCYSBI(tm) 25mg and
75mg gastro-resistant hard capsules, cysteamine (as mercaptamine bitartrate)
for the treatment of proven nephropathic cystinosis. If approved, PROCYSBI will
be indicated for the treatment of proven nephropathic cystinosis.

The positive opinion from CHMP must be ratified by the European Commission (EC)
in order to grant marketing authorization for PROCYSBI, which would cover all
27 European Union member countries including Iceland and Norway. A decision is
expected from the European Commission within a few months of the CHMP
recommendation.

'The positive opinion of the CHMP brings us an important step closer to
anticipated EU approval of PROCYSBI subject to the European Commission review
process,' said Christopher M. Starr, Ph.D., Raptor's chief executive officer.
'While this recommendation is an important milestone for Raptor, it is also
good news for European patients who suffer from nephropathic cystinosis.'

PROCYSBI is a new therapy for the management of nephropathic cystinosis that is
taken orally every twelve hours. PROCYSBI was engineered to bypass the stomach
with an extended terminal half-life so that patients experience steady drug
levels in their bodies for the full 12-hour dosing period. Randomized
controlled clinical trials and extended treatment with PROCYSBI therapy
demonstrated consistent and continuous control of white blood cell cystine.

U.S. Product Information about PROCYSBI (cysteamine bitartrate)



PROCYSBI is a cystine depleting agent that is approved in the U.S. for the
management of nephropathic cystinosis in adults and children ages 6 years and
older. It is contraindicated in patients with a hypersensitivity to
penicillamine. The most commonly reported side effects are vomiting, abdominal
pain/discomfort, headaches, nausea, diarrhea, anorexia/decreased appetite,
breath odor, fatigue, dizziness, skin odor, and rash.

If European Commission approval is obtained, the indications,
contraindications, warnings and precautions ultimately adopted by the European
Commission may be different from those in the U.S.

Important U.S. Safety Information about PROCYSBI

-- Patients should be monitored for development of skin or bone lesions and
dosage of PROCYSBI reduced as needed.
-- If a severe skin rash develops such as erythema multiforme bullosa or toxic
epidermal necrolysis, PROCYSBI should be discontinued.
-- Cysteamine has been associated with gastrointestinal ulceration and
bleeding.
-- Central Nervous System (CNS) symptoms such as seizures, lethargy,
somnolence, depression, and encephalopathy have been associated with
immediate-release cysteamine. Patients should exercise caution in driving a
car or engaging in other hazardous activities after taking PROCYSBI.
-- Cysteamine has been associated with reversible leukopenia and abnormal
liver function studies. Therefore, blood counts and liver function tests
should be monitored.
-- Benign intracranial hypertension (or pseudotumor cerebri PTC) and/or
papilledema has been associated with immediate-release cysteamine
bitartrate treatment. Physicians should monitor for signs and symptoms of
PTC.
-- Breastfeeding is not recommended for nursing mothers taking PROCYSBI.

For additional information on PROCYSBI, including full prescribing information,
please visit www.raptorpharma.com.

About Nephropathic Cystinosis

Nephropathic cystinosis comprises 95% of cases of cystinosis, a rare,
life-threatening metabolic lysosomal storage disorder that causes toxic
accumulation of cystine in all cells, tissues, and organs in the body. Elevated
cystine leads to progressive, irreversible tissue damage and multi-organ
failure, including kidney failure, blindness, muscle wasting and premature
death. Nephropathic cystinosis is usually diagnosed in infancy and requires
lifelong therapy. Left untreated, the disease is usually fatal by the end of
the first decade of life. There are an estimated 500 patients living in the
U.S. with cystinosis, and 2,000 worldwide.

Cystine depletion is the primary treatment strategy for nephropathic
cystinosis. However, poor adherence to therapy has been a major challenge
resulting in poor sustained control of cystine levels, and patients
consequently experience poor clinical outcomes, including kidney insufficiency
leading to dialysis and kidney transplantation, muscle wasting and in some
cases, premature death. Even brief interruptions in daily therapy can permit
toxic accumulation of cystine, exposing tissues to renewed, progressive
deterioration.

About Raptor Pharmaceutical

Raptor Pharmaceutical Corp. is a biopharmaceutical company focused on
developing and commercializing life-altering therapeutics that treat rare,
debilitating and often fatal diseases. The company's first product, PROCYSBI(tm)
(cysteamine bitartrate) delayed-release capsules, has been approved by the FDA
and is currently being reviewed by the European Commission, following a
positive recommendation from the CHMP, as a potential new treatment for
nephropathic cystinosis, a rare metabolic lysosomal storage disease. Raptor's
pipeline also includes RP103 in a Phase 2/3 trial for Huntington's disease and
a Phase 2 trial in nonalcoholic fatty liver disease in children. For additional
information, please visit www.raptorpharma.com.

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 operations or future financial
performance, including, but not limited to statements regarding anticipated EU
approval of PROCYSBI and the timing of that anticipated approval. 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 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 transition
report for the four months ended December 31, 2012 on Form 10-KT filed with the
SEC on March 14, 2013, as amended by the Form 10-KT/A filed with the SEC on
June 19, 2013,and Raptor's Quarterly Report on Form 10-Q filed with the SEC on
May 8, 2013, as amended by the Form 10-Q/A filed with the SEC on June 19, 2013,
which are available free of charge on the SEC's website at 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: Georgia Erbez
Chief Financial Officer
Raptor Pharmaceutical Corp.
(415) 382-8111 x204
gerbez(at)raptorpharma.com

INVESTOR CONTACTS:
Westwicke Partners, LLC
Stefan Loren, Ph.D.
Managing Director
(443) 213-0507
sloren(at)westwicke.com
Robert H. Uhl
Managing Director
(858) 356-5932
robert.uhl(at)westwicke.com

MEDIA CONTACT:
Carolyn Hawley
Canale Communications
(619) 849-5375
carolyn(at)canalecomm.com
News Source: NASDAQ OMX



28.06.2013 Dissemination of a Corporate News, transmitted by DGAP -
a company of EQS Group AG.
The issuer is solely responsible for the content of this announcement.

DGAP's Distribution Services include Regulatory Announcements,
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Language: English
Company: Raptor Pharmaceutical Corp.


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Date: 06/28/2013 - 08:58
Language: English
News-ID 1241380
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