Raptor Pharmaceutical firm.Press releaseRaptor Pharmaceutical's PROCYSBI(TM) Receives Positive Recommendation From CHMPNOVATO, Calif., 2013-06-28 14:58 CEST (GLOBE NEWSWIRE) -- bird Pharmaceutical
Corp. (Nasdaq:RPTP) these days declared these days that the ecu Committee for
Medicinal product for Human Use (CHMP) has adopted a positive opinion
recommending selling authorization for PROCYSBI(TM) 25mg and 75mg
gastro-resistant arduous capsules, cysteamine (as mercaptamine bitartrate) for the
treatment of tested nephropathic cystinosis. If approved, PROCYSBI are
indicated for the treatment of tested nephropathic cystinosis.
The positive opinion from CHMP should be legal by the ecu Commission (EC)
in order to grant selling authorization for PROCYSBI, which might cowl all
27 world organization member countries together with Iceland and Norge. a choice is
expected from the ecu Commission inside a couple of months of the CHMP
recommendation.
"The positive opinion of the CHMP brings America a crucial step nearer to
anticipated EU approval of PROCYSBI subject to the ecu Commission review
process," same Christopher M. Starr, Ph.D., Raptor's chief officer.
"While this recommendation is a crucial milestone for bird, it is also
good news for European patients that suffer from nephropathic cystinosis."
PROCYSBI may be a new medical aid for the management of nephropathic cystinosis that's
taken orally each twelve hours. PROCYSBI was built to bypass the abdomen
with associate extended terminal half-life so patients expertise steady drug
levels in their bodies for the complete 12-hour dosing amount. Randomized
controlled clinical trials and extended treatment with PROCYSBI medical aid
demonstrated consistent and continuous management of white corpuscle amino acid.
U.S. Product info concerning PROCYSBI (cysteamine bitartrate)
PROCYSBI may be a amino acid depleting agent that's approved within the U.S. for the
management of nephropathic cystinosis in adults and youngsters ages six years and
older. it's contraindicated in patients with a hypersensitivity to
penicillamine. the foremost normally reportable aspect effects square measure forcing out, abdominal
pain/discomfort, headaches, nausea, diarrhea, anorexia/decreased appetence,
breath odor, fatigue, dizziness, skin odor, and rash.
If European Commission approval is obtained, the indications,
contraindications, warnings and precautions ultimately adopted by the ecu
Commission is also completely different from those within the U.S.
Important U.S. Safety info concerning PROCYSBI
-- Patients ought to be monitored for development of skin or bone lesions and
dose of PROCYSBI reduced PRN.
-- If a severe efflorescence develops like erythema bullosa or ototoxic
dermal lysis, PROCYSBI ought to be out of print.
-- Cysteamine has been related to canal ulceration and
bleeding.
-- Central system (CNS) symptoms like seizures, lethargy,
somnolence, depression, and neurological disease are related to
immediate-release cysteamine. Patients ought to exercise caution in driving a
automobile or partaking in alternative venturesome activities when taking PROCYSBI.
-- Cysteamine has been related to reversible blood disease and abnormal
liver perform studies. Therefore, blood counts and liver perform tests
ought to be monitored.
-- Benign intracranial cardiovascular disease (or pseudotumor cerebri PTC) and/or
edema has been related to immediate-release cysteamine
tartrate treatment. Physicians ought to monitor for signs and symptoms of
PTC.
-- Breastfeeding isn't suggested for nursing mothers taking PROCYSBI.
For additional info on PROCYSBI, together with full prescribing info,
please visit WWW.raptorpharma.com.
About Nephropathic Cystinosis
Nephropathic cystinosis includes ninety fifth of cases of cystinosis, a rare,
life-threatening metabolic lysosomal storage disorder that causes ototoxic
accumulation of amino acid altogether cells, tissues, and organs within the body. Elevated
cystine ends up in progressive, irreversible tissue injury and multi-organ
failure, together with nephropathy, blindness, muscle wasting and premature
death. Nephropathic cystinosis is sometimes diagnosed in infancy and needs
lifelong medical aid. Left untreated, the illness is sometimes fatal by the top of
the first decade of life. There square measure associate calculable five hundred patients living within the
U.S. with cystinosis, and 2,000 worldwide.
Cystine depletion is that the primary treatment strategy for nephropathic
cystinosis. However, poor adherence to medical aid has been a serious challenge
resulting in poor sustained management of amino acid levels, and patients
consequently expertise poor clinical outcomes, together with excretory organ insufficiency
leading to chemical analysis and excretory organ transplantation, muscle wasting and in some
cases, premature death. Even temporary interruptions in daily medical aid will allow
toxic accumulation of amino acid, exposing tissues to revived, progressive
deterioration.
About bird Pharmaceutical
Raptor Pharmaceutical firm. may be a biopharmaceutical company targeted on
developing and commercializing life-altering medicine that treat rare,
debilitating and sometimes fatal diseases. The company's initial product, PROCYSBI(TM)
(cysteamine bitartrate) delayed-release capsules, has been approved by the Food and Drug Administration
and is presently being reviewed by the ecu Commission, following a
positive recommendation from the CHMP, as a possible new treatment for
nephropathic cystinosis, a rare metabolic lysosomal storage illness. Raptor's
pipeline additionally includes RP103 during a section 2/3 trial for chorea and
a section a pair of trial in soft {fatty liver|liver illness} disease in kids. for extra
information, please visit WWW.raptorpharma.com.
Forward trying Statements
This document contains advanced statements as that term is outlined in
the non-public Securities proceedings Reform Act of 1995. These statements relate
to future events or our future results of operations or future monetary
performance, including, however not restricted to statements concerning anticipated EU
approval of PROCYSBI and also the temporal order of that anticipated approval. These
statements square measure solely predictions and involve noted and unknown risks,
uncertainties and alternative factors, which can cause the Company's actual results
to be materially completely different from these advanced statements. Factors that
may considerably amendment or stop the Company's forward trying statements
from fruition square measure delineated in larger detail within the Company's filings from
time to time with the Securities and Exchange Commission (the "SEC"), which
Raptor powerfully urges you to browse and contemplate, including: Raptor's transition
report for the four months complete day, 2012 on kind 10-KT filed with the
SEC on March fourteen, 2013, as amended by the shape 10-KT/A filed with the SEC on
June 19, 2013,and Raptor's Quarterly Report on kind 10-Q filed with the SEC on
May 8, 2013, as amended by the shape 10-Q/A filed with the SEC on June nineteen, 2013,
which square measure out there freed from charge on the SEC's web site at WWW.sec.gov.
Subsequent written and oral advanced statements as a result of bird
or to persons working on its behalf square measure expressly qualified in their totality 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.
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