SAN DIEGO, CA and LEIDEN, THE NETHERLANDS--(Marketwired - June 18, 2013) - Santarus, Inc.
(NASDAQ: SNTS) and Pharming Group NV (NYSE Euronext: PHARM) today announced
that the U.S.
Food and Drug Administration (FDA) has accepted for filing the Biologics
Application (BLA) for the investigational drug RUCONEST® (recombinant
esterase inhibitor) 50 IU/kg. Santarus and Pharming are seeking U.S.
approval of RUCONEST for the treatment of acute angioedema attacks in
with hereditary angioedema (HAE). The FDA indicated that as part of its
it plans to present the BLA to the Blood Products Advisory Committee.
to the Prescription Drug User Fee Act (PDUFA) guidelines, Santarus and
expect the FDA will complete its review or otherwise respond to the
by April 16, 2014.
The safety and efficacy of RUCONEST for the treatment of HAE attacks were
evaluated in a clinical program that included a Phase III randomized
placebo-controlled study conducted under a Special Protocol Assessment
the FDA. The RUCONEST clinical program also included two additional
placebo-controlled studies and several open label treatment studies.
"RUCONEST is the first recombinant C1 esterase inhibitor developed with the
of treating the pain and swelling associated with acute HAE attacks," said
Gerald T. Proehl, president and chief executive officer of Santarus. "We
believe RUCONEST has the potential to be an important new therapeutic
patients experiencing acute HAE attacks based on the data contained in the
from ten clinical studies covering 940 administrations of the drug."
"Acceptance of the BLA is a pivotal event for Pharming and represents the
significant step to date in our efforts to obtain marketing approval for
RUCONEST in the U.S.," said Sijmen de Vries, chief executive officer of
Pharming. "We look forward to working with our colleagues at Santarus to
RUCONEST through the U.S. regulatory process, and ultimately provide a new
therapy to physicians and the patients they treat."
Santarus licensed certain exclusive rights from Pharming to commercialize
RUCONEST in North America for the treatment of acute attacks of HAE as well
other potential future indications. Under the terms of the license
$5 million milestone is payable to Pharming as a result of the FDA
for review of the BLA for RUCONEST.
About RUCONEST and Hereditary Angioedema
RUCONEST (INN conestat alfa) is a recombinant version of the human protein
esterase inhibitor, and is produced with Pharming's proprietary transgenic
technology. RUCONEST is approved in Europe for the treatment of acute
attacks in patients with HAE, a genetic disorder in which the patient is
deficient in or lacks a functional plasma protein C1 esterase inhibitor,
resulting in unpredictable and debilitating episodes of intense swelling.
swelling may occur in one or more anatomical areas, including the
face, trunk, genitals, abdomen and upper airway. The frequency and severity
HAE attacks vary and are most serious when they involve laryngeal edema,
can close the upper airway and cause death by asphyxiation. According to
U.S. Hereditary Angioedema Association, epidemiological estimates for HAE
from one in 10,000 to one in 50,000 individuals. RUCONEST is an
drug in the U.S. and has been granted orphan drug designation by the FDA
for the treatment of acute attacks of HAE and for prophylactic treatment of
About Pharming Group NV
Pharming Group NV is developing innovative products for the treatment of
medical needs. RUCONEST® is a recombinant human C1 esterase inhibitor
for the treatment of angioedema attacks in patients with HAE in all 27 EU
countries plus Norway, Iceland and Liechtenstein, and is distributed in the
by Swedish Orphan Biovitrum. RUCONEST® is partnered with Santarus,
Inc. (NASDAQ: SNTS) in North America and a Biologics License Application for RUCONEST is
review by the U.S. Food and Drug Administration. The product is also being
evaluated for various follow-on indications. Pharming has a unique GMP
compliant, validated platform for the production of recombinant human
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. Pharming now plans to utilise this platform for the
development of rhFVIII for the treatment of Haemophilia A. Additional
information is available on the Pharming website, www.pharming.com.
Santarus, Inc. is a specialty biopharmaceutical company focused on
developing and commercializing proprietary products that address the needs
patients treated by physician specialists. The company's current commercial
efforts are focused on five products. UCERIS® (budesonide) extended
tablets for the induction of remission in patients with active, mild to
ulcerative colitis and ZEGERID® (omeprazole/sodium bicarbonate) for
treatment of certain upper gastrointestinal disorders are promoted to
gastroenterologists. GLUMETZA® (metformin hydrochloride extended
tablets) and CYCLOSET® (bromocriptine mesylate) tablets, which are
adjuncts to diet and exercise to improve glycemic control in adults with
diabetes, and FENOGLIDE® (fenofibrate) tablets, which is indicated as
to diet to reduce high cholesterol, are promoted to endocrinologists and
physicians who treat patients with type 2 diabetes. Full prescribing and
information for Santarus' products is available at www.santarus.com or by
contacting Santarus at 1-888-778-0887.
Santarus' product development pipeline includes the investigational drug
RUCONEST® (recombinant human C1 esterase inhibitor). A Biologics
Application for RUCONEST for the treatment of acute angioedema attacks in
patients with hereditary angioedema is under review by the U.S Food and
Administration with a response expected in April 2014. Santarus is also
developing rifamycin SV MMX®, which is in Phase III clinical testing
treatment of travelers' diarrhea. In addition, the company has completed a
Phase I clinical program with SAN-300, an investigational monoclonal
More information about Santarus is available at www.santarus.com.
Santarus and Pharming caution you that statements included in this press
that are not a description of historical facts are forward-looking
The inclusion of forward-looking statements should not be regarded as a
representation by Santarus or Pharming that any of its plans or objectives
be achieved. Actual results may differ materially from those set forth in
release due to the risks and uncertainties inherent in Santarus and
businesses, including, without limitation: whether the FDA will approve
RUCONEST BLA in a timely manner or at all; whether the FDA will concur with
clinical interpretation of the Phase III study results or the conduct of
study; whether the FDA ultimately will require additional clinical studies
other development programs before approving RUCONEST; risks related to
dependence on Pharming for many functions related to RUCONEST, and
ability to continue to perform these functions based on its limited
resources; risks related to the license and supply arrangements between
and Pharming, including the potential for termination of the arrangements;
difficulties or delays in development, testing, manufacturing and marketing
and obtaining and maintaining regulatory approvals for, Santarus and
products; and other risks detailed in prior press releases as well as in
periodic filings with the Securities and Exchange Commission, including
Santarus' Quarterly Report on Form 10-Q for the quarter ended March 31,
You are cautioned not to place undue reliance on these forward-looking
statements, which speak only as of the date hereof. All forward-looking
statements are qualified in their entirety by this cautionary statement and
neither Santarus nor Pharming undertakes any obligation to revise or update
news release to reflect events or circumstances after the date hereof,
may be required by law. This caution is made under the safe harbor
Section 21E of the Private Securities Litigation Reform Act of 1995.
Santarus®, FENOGLIDE®, UCERIS®, and ZEGERID® are
registered trademarks of
Santarus, Inc. GLUMETZA® is a trademark of Biovail Laboratories
S.r.l. licensed exclusively in the United States to Depomed, Inc.
CYCLOSET® is a
trademark of VeroScience LLC. MMX® is a trademark of Cosmo
RUCONEST® is a trademark of Pharming Group N.V.
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Source: Pharming Group N.V. via Thomson Reuters ONE