Santarus and Pharming announce FDA acceptance for review of RUCONEST (recombinant human C1 esterase inhibitor) Biologics License Application
(Thomson Reuters ONE) -
SAN DIEGO & Leiden, The Netherlands (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 License
Application (BLA) for the investigational drug RUCONEST® (recombinant human C1
esterase inhibitor) 50 IU/kg. Santarus and Pharming are seeking U.S. marketing
approval of RUCONEST for the treatment of acute angioedema attacks in patients
with hereditary angioedema (HAE). The FDA indicated that as part of its review
it plans to present the BLA to the Blood Products Advisory Committee. Pursuant
to the Prescription Drug User Fee Act (PDUFA) guidelines, Santarus and Pharming
expect the FDA will complete its review or otherwise respond to the RUCONEST BLA
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 agreement with
the FDA. The RUCONEST clinical program also included two additional randomized
placebo-controlled studies and several open label treatment studies.
"RUCONEST is the first recombinant C1 esterase inhibitor developed with the goal
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 option for
patients experiencing acute HAE attacks based on the data contained in the BLA
from ten clinical studies covering 940 administrations of the drug."
"Acceptance of the BLA is a pivotal event for Pharming and represents the most
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 move
RUCONEST through the U.S. regulatory process, and ultimately provide a new HAE
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 as
other potential future indications. Under the terms of the license agreement, a
$5 million milestone is payable to Pharming as a result of the FDA acceptance
for review of the BLA for RUCONEST.
About RUCONEST and Hereditary Angioedema
RUCONEST (INN conestat alfa) is a recombinant version of the human protein C1
esterase inhibitor, and is produced with Pharming's proprietary transgenic
technology. RUCONEST is approved in Europe for the treatment of acute angioedema
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. The
swelling may occur in one or more anatomical areas, including the extremities,
face, trunk, genitals, abdomen and upper airway. The frequency and severity of
HAE attacks vary and are most serious when they involve laryngeal edema, which
can close the upper airway and cause death by asphyxiation. According to the
U.S. Hereditary Angioedema Association, epidemiological estimates for HAE range
from one in 10,000 to one in 50,000 individuals. RUCONEST is an investigational
drug in the U.S. and has been granted orphan drug designation by the FDA both
for the treatment of acute attacks of HAE and for prophylactic treatment of HAE.
About Pharming Group NV
Pharming Group NV is developing innovative products for the treatment of unmet
medical needs. RUCONEST® is a recombinant human C1 esterase inhibitor approved
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 EU
by Swedish Orphan Biovitrum. RUCONEST® is partnered with Santarus, Inc. (NASDAQ:
SNTS) in North America and a Biologics License Application for RUCONEST is under
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 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. 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.
About Santarus
Santarus, Inc. is a specialty biopharmaceutical company focused on acquiring,
developing and commercializing proprietary products that address the needs of
patients treated by physician specialists. The company's current commercial
efforts are focused on five products. UCERIS® (budesonide) extended release
tablets for the induction of remission in patients with active, mild to moderate
ulcerative colitis and ZEGERID® (omeprazole/sodium bicarbonate) for the
treatment of certain upper gastrointestinal disorders are promoted to
gastroenterologists. GLUMETZA® (metformin hydrochloride extended release
tablets) and CYCLOSET® (bromocriptine mesylate) tablets, which are indicated as
adjuncts to diet and exercise to improve glycemic control in adults with type 2
diabetes, and FENOGLIDE® (fenofibrate) tablets, which is indicated as an adjunct
to diet to reduce high cholesterol, are promoted to endocrinologists and other
physicians who treat patients with type 2 diabetes. Full prescribing and safety
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 License
Application for RUCONEST for the treatment of acute angioedema attacks in
patients with hereditary angioedema is under review by the U.S Food and Drug
Administration with a response expected in April 2014. Santarus is also
developing rifamycin SV MMX®, which is in Phase III clinical testing for the
treatment of travelers' diarrhea. In addition, the company has completed a
Phase I clinical program with SAN-300, an investigational monoclonal antibody.
More information about Santarus is available at www.santarus.com.
Santarus and Pharming caution you that statements included in this press release
that are not a description of historical facts are forward-looking statements.
The inclusion of forward-looking statements should not be regarded as a
representation by Santarus or Pharming that any of its plans or objectives will
be achieved. Actual results may differ materially from those set forth in this
release due to the risks and uncertainties inherent in Santarus and Pharming's
businesses, including, without limitation: whether the FDA will approve the
RUCONEST BLA in a timely manner or at all; whether the FDA will concur with the
clinical interpretation of the Phase III study results or the conduct of the
study; whether the FDA ultimately will require additional clinical studies or
other development programs before approving RUCONEST; risks related to Santarus'
dependence on Pharming for many functions related to RUCONEST, and Pharming's
ability to continue to perform these functions based on its limited financial
resources; risks related to the license and supply arrangements between Santarus
and Pharming, including the potential for termination of the arrangements; other
difficulties or delays in development, testing, manufacturing and marketing of,
and obtaining and maintaining regulatory approvals for, Santarus and Pharming's
products; and other risks detailed in prior press releases as well as in public
periodic filings with the Securities and Exchange Commission, including
Santarus' Quarterly Report on Form 10-Q for the quarter ended March 31, 2013.
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 this
news release to reflect events or circumstances after the date hereof, except as
may be required by law. This caution is made under the safe harbor provisions of
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 International
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 Technologies Limited.
RUCONEST® is a trademark of Pharming Group N.V.
# # #
SANTARUS CONTACTS: PHARMING CONTACTS:
Martha L. Hough, VP Finance & Investor Relations Sijmen de Vries, CEO
+1 (858) 314-5824 Tel: +31 (0)71 524 7400
Debra P. Crawford, Chief Financial Officer
+1 (858) 314-5708
INVESTOR CONTACTS:
Westwicke Partners, LLC FTI Consulting
Stefan Loren, Ph.D. (sloren(at)westwicke.com) Julia Phillips/John Dineen
+1 (858) 356-5930 Tel: +44 (0)207 269 7193
Robert Uhl (robert.uhl(at)westwicke.com)
+1 (858) 356-5932
Press release (PDF):
http://hugin.info/132866/R/1710102/566919.pdf
This announcement is distributed by Thomson Reuters on behalf of
Thomson Reuters clients. The owner of this announcement warrants that:
(i) the releases contained herein are protected by copyright and
other applicable laws; and
(ii) they are solely responsible for the content, accuracy and
originality of the information contained therein.
Source: Pharming Group N.V. via Thomson Reuters ONE
[HUG#1710102]
Unternehmensinformation / Kurzprofil:
Datum: 18.06.2013 - 07:00 Uhr
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News-ID 270520
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