LEIDEN, The Netherlands and
RALEIGH, North Carolina,
November 3, 2014 /PRNewswire/ --
RUCONEST® (C1
esterase inhibitor [recombinant]) 50 iu/kg is the first and only
recombinant treatment option available in the U.S. for adult and
adolescent patients suffering from HAE
Pharming Group NV (EURONEXT: PHARM) and Salix Pharmaceuticals,
Ltd. (NASDAQ:SLXP) today announced the launch of
RUCONEST® (C1 Esterase Inhibitor [Recombinant]) 50
IU/kg in the United States for the
treatment of acute angioedema attacks in adult and adolescent
patients with hereditary angioedema (HAE). Effectiveness in
clinical studies was not established in HAE patients with laryngeal
attacks. Today's announcement follows the July approval of the drug
by the Food and Drug Administration.
"We're excited to offer the only recombinant C1 esterase
inhibitor therapy for HAE in the United
States," said Carolyn J.
Logan, President and Chief Executive Officer of Salix.
"RUCONEST treats the root cause of HAE attacks, which has been
shown to raise C1 inhibitor levels to within the normal range.
RUCONEST can be self-administered by appropriately trained patients
and is effective at stopping most HAE attacks in one dose."
RUCONEST, a recombinant C1 esterase inhibitor, can be
administered by the patient after receiving training by a
healthcare provider. RUCONEST is over 98 percent pure, and
because it is not made from human plasma, it does not carry any
known risk of passing on viruses that can be found in human
blood.
HAE is a rare genetic condition that affects between 1 in 10,000
to 1 in 50,000 people. It causes episodes of swelling in various
parts of the body, including the hands, feet, abdomen and face.
Patients with abdominal swelling often experience severe pain,
nausea and vomiting. HAE attacks stem from a deficiency of the C1
inhibitor protein in the blood. The disease is often misdiagnosed,
as the symptoms of an attack can mirror someone experiencing an
allergic reaction. Severe, painful swelling can occur at any time,
which means most people suffering from HAE deal with the constant
fear of when their next attack might surface and how that might
impair their lives and those around them.
"HAE is an especially challenging disease for patients to
manage," said Anthony Castaldo,
President of the Hereditary Angioedema Association (US HAEA), a
non-profit patient services and research organization with a
membership of over 5,000 HAE patients in the United States. "If left untreated,
patients can experience attacks that are incredibly painful and,
because of its unpredictability, HAE interferes with daily life.
We're pleased HAE patients now have another treatment option
available to them."
RUCONEST is available by prescription across the United States through RUCONEST SOLUTIONS
and comes with comprehensive patient support services. For
more information, including an opportunity for a free trial of
RUCONEST, visit Ruconest.com or call RUCONEST® SOLUTIONS
at (855) 613-4HAE.
RUCONEST is manufactured by Pharming Group NV in the Netherlands. Salix has licensed exclusive
rights from Pharming to commercialize
RUCONEST® in North America and market RUCONEST for the
treatment of acute HAE attack symptoms.
"RUCONEST's availability in the United
States marks a significant milestone for Pharming," said
Sijmen de Vries, CEO of Pharming. "RUCONEST has helped patients in
other countries around the world and we look forward to seeing the
difference it will make in the lives of HAE patients in the
U.S."
Indication:
RUCONEST® is a C1 esterase inhibitor [recombinant]
indicated for the treatment of acute attacks in adult and
adolescent patients with hereditary angioedema (HAE). Effectiveness
in clinical studies was not established in HAE patients with
laryngeal attacks.
Important Safety Information for RUCONEST
RUCONEST® (C1 esterase inhibitor [recombinant]) is
contraindicated in patients with a history of allergy to rabbits or
rabbit-derived products, and patients with a history of
life-threatening immediate hypersensitivity reactions to C1
esterase inhibitor preparations, including anaphylaxis.
Severe hypersensitivity reactions may occur. The signs and
symptoms of hypersensitivity reactions may include hives,
generalized urticaria, tightness of the chest, wheezing,
hypotension, and/or anaphylaxis during or after injection of
RUCONEST. Should symptoms occur, discontinue RUCONEST and
administer appropriate treatment. Because hypersensitivity
reactions may have symptoms similar to HAE attacks, treatment
methods should be carefully considered.
Serious arterial and venous thromboembolic (TE) events have been
reported at the recommended dose of plasma derived C1 esterase
inhibitor products in patients with risk factors. Risk factors may
include the presence of an indwelling venous catheter/access
device, prior history of thrombosis, underlying atherosclerosis,
use of oral contraceptives or certain androgens, morbid obesity,
and immobility. Monitor patients with known risk factors for
TE events during and after RUCONEST administration.
RUCONEST has not been studied in pregnant women; therefore, it
should only be used during pregnancy if clearly needed.
The most common adverse reactions (incidence ≥2%) were headache,
nausea, and diarrhea. The serious adverse reaction in clinical
studies of RUCONEST was anaphylaxis.
Please see complete Prescribing Information for
RUCONEST.
About RUCONEST®
RUCONEST®(C1 Esterase Inhibitor [Recombinant]) 50
IU/kg is an injectable medicine that is used to treat acute
angioedema attacks in adult and adolescent patients with hereditary
angioedema (HAE). HAE is caused by a deficiency of the C1
esterase inhibitor protein, which is present in blood and helps
control inflammation (swelling) and parts of the immune system. A
shortage of C1 esterase inhibitor can lead to repeated attacks of
swelling, pain in the abdomen, difficulty breathing and other
symptoms. RUCONEST®contains C1 esterase inhibitor at 50
IU/kg.
When administered at the onset of HAE attack symptoms at the
recommended dose, RUCONEST works to return a patient's C1-INH
levels to normal range and quickly begins to relieve the symptoms
of an HAE attack with a low recurrence of symptoms.
RUCONEST is the first and only plasma-free, recombinant
C1-INH approval from the U.S. Food and Drug Administration (FDA)
and was approved in July 2014.
RUCONEST has been granted Orphan Drug designation by the FDA for
the treatment of acute angioedema attacks in patients with
hereditary angioedema (HAE). With RUCONESTnow approved by the
FDA, Salix believes this designation should provide seven years of
marketing exclusivity in the United
States.
About HAE
Hereditary angioedema (HAE) is a genetic condition occurring
between 1 in 10,000 to 1 in 50,000 people. Those with HAE
experience episodes of swelling in their extremities, face and
abdomen, with potentially life-threatening swelling of the airway.
When it occurs in the abdomen, this swelling can be accompanied by
bouts of nausea, vomiting and severe pain. Swelling in the face or
extremities can be painful, disfiguring, and disabling.
HAE patients have a defect in the gene that controls production
of a protein found in the blood vessels, called C1 inhibitor or
C1-INH. When a person's C1-INH levels are low, fluid from blood
vessels can leak into nearby connective tissues, causing severe
pain and swelling and, in rare cases, death from asphyxiation from
airway swelling.
About Pharming Group NV
Pharming Group NV is developing innovative products for the
treatment of unmet medical needs. Ruconest® (conestat
alfa) is a recombinant human C1 esterase inhibitor approved for the
treatment of angioedema attacks in patients with HAE in the
USA, Israel, all 27 EU countries plus Norway, Iceland and Liechtenstein. Ruconest is commercialized by
Pharming in Austria, Germany and Netherlands. Ruconest is distributed by
Swedish Orphan Biovitrum AB (publ) (SS: SOBI) in the other EU
countries and in Azerbaijan,
Belarus, Georgia, Iceland, Kazakhstan, Liechtenstein, Norway, Russia, Serbia and Ukraine.
Ruconest is partnered with Salix Pharmaceuticals Inc. (NASDAQ:
SLXP) in North America.
Ruconest is also being investigated in a randomized Phase II
clinical trial for prophylaxis of HAE and evaluated for various
additional follow-on indications. Pharming has a unique GMP
compliant, validated platform for the production of recombinant
human proteins that has proven capable of producing industrial
volumes of high quality recombinant human protein in a more
economical way compared to current cell based technologies. Leads
for enzyme replacement therapy in Pompe's, Fabry's and Gaucher's
diseases are under early evaluation. The platform is partnered with
Shanghai Institute for Pharmaceutical Industry (SIPI), a Sinopharm
Company, for joint global development of new products. Pre-
clinical development and manufacturing will take place at SIPI and
are funded by SIPI. Pharming and SIPI initially plan to utilize
this platform for the development of rh-FVIII for the treatment of
Haemophilia-A. Additional information is available on the Pharming
website; http://www.pharming.com .
About Salix Pharmaceuticals
Salix Pharmaceuticals, Ltd., headquartered in Raleigh, North Carolina, develops and markets
prescription pharmaceutical products and medical devices for the
prevention and treatment of gastrointestinal diseases. Salix's
strategy is to in-license late-stage or marketed proprietary
therapeutic products, complete any required development and
regulatory submission of these products, and commercialize them
through the Company's 500-member specialty sales force.
Salix trades on the NASDAQ Global Select Market under the ticker
symbol "SLXP". For more information, please visit our website
at http://www.salix.com or contact Salix at
919-862-1000. Follow us on Twitter (@SalixPharma) and Facebook
(http://www.facebook.com/SalixPharma). Information on our Twitter
feed, Facebook page and website is not incorporated in our filings
with the SEC.
Salix Disclosure Notice
Please Note: The statements provided herein that are not
historical facts are or might constitute projections and other
forward-looking statements regarding future events. Although
we believe the expectations reflected in such forward-looking
statements are based on reasonable assumptions, our expectations
might not be attained. Forward-looking statements are just
predictions and are subject to known and unknown risks and
uncertainties that could cause actual events or results to differ
materially from expected results. Factors that could cause
actual events or results to differ materially from those described
herein include, among others: uncertainty that Ruconest will be
commercially successful; market acceptance for approved products;
generic and other competition in an increasingly global industry;
litigation and the possible impairment of, or inability to obtain,
intellectual property rights and the costs of obtaining such rights
from third parties in an increasingly global
industry; the unpredictability of the duration and
results of regulatory review of New Drug Applications, Biologics
License Agreements and Investigational NDAs, including risk that
XIFAXAN (rifaximin) 550 mg will not receive the necessary
regulatory approvals for Irritable Bowel Syndrome with
Diarrhea; the cost, timing and results of clinical
trials and other development activities involving pharmaceutical
products; post-marketing approval regulation, including the ongoing
Department of Justice investigation of Salix's marketing practices;
revenue recognition and other critical accounting policies; the
need to acquire new products; changes in tax laws or
interpretations thereof; general economic and business conditions;
and other factors. Readers are cautioned not to place undue
reliance on the forward-looking statements included herein, which
speak only as of the date hereof. Salix does not undertake to
update any of these statements in light of new information or
future events, except as required by law. The reader is
referred to the documents that Salix files from time to time with
the SEC.
Contacts:
Pharming Group NV
Sijmen de Vries
Chief Executive Officer
+31-71-5247400
Julia Phillips/ Victoria Foster Mitchell
FTI Consulting
+44-203-727-1136
Salix Pharmaceuticals
Adam C. Derbyshire
Executive Vice President and Chief Financial Officer
+1-919-862-1000
G. Michael Freeman
Associate Vice President, Investor Relations and Corporate
Communications
+1-919-862-1000
PRN NLD
