European Medicines Agency Validates Marketing Authorization Application for Natpar® (parathyroid hormone (rDNA)) in Hypopara...
December 02 2014 - 9:16AM
Business Wire
NPS Pharmaceuticals, Inc. (NASDAQ:NPSP), a global
biopharmaceutical company pioneering and delivering therapies that
transform the lives of patients with rare diseases, today announced
that the European Medicines Agency (EMA) has validated and
initiated its review of the company’s marketing authorization
application (MAA) for Natpar® (parathyroid hormone (rDNA)) for the
treatment of Hypoparathyroidism, a rare endocrine disorder
characterized by insufficient levels of parathyroid hormone (PTH).
If approved, Natpar would be the first and only PTH replacement
therapy in Europe for patients with Hypoparathyroidism.
“MAA validation for Natpar is an important achievement for our
emerging global endocrine franchise,” said Francois Nader, MD,
president and chief executive officer of NPS Pharma. “Without an
approved replacement therapy, the current goal of
Hypoparathyroidism management is limited to symptom control, which
does not address the underlying cause of the disorder. In clinical
studies, Natpar has demonstrated similar physiologic effects to the
native parathyroid hormone, showing promise as long-term PTH
replacement therapy.”
The MAA includes results from 13 pharmacology studies and four
company-sponsored efficacy and safety studies. The pivotal Phase 3
study, known as REPLACE, was a multinational, randomized,
double-blind, placebo controlled study and the largest clinical
trial conducted to date in Hypoparathyroidism. Key findings from
the study were published in The Lancet Diabetes and Endocrinology
(2013; 1:275-283).
The EMA granted orphan drug designation for Natpar for the
treatment of Hypoparathyroidism in January 2014. The U.S. Food and
Drug Administration (FDA) granted orphan drug status for the
product for the treatment of Hypoparathyroidism in 2007 and a
Biologics License Application to market it under the trade name
Natpara® is currently under review by the FDA with a Prescription
Drug User Fee Act action date of January 24, 2015.
About Hypoparathyroidism
Hypoparathyroidism is a rare condition in which the parathyroid
glands fail to produce sufficient amounts of parathyroid hormone
(PTH) or where PTH lacks biologic activity. PTH plays a central
role in a variety of critical physiological functions in the body.
In patients with Hypoparathyroidism, insufficient levels of PTH
lead to many physiological abnormalities, including low serum
calcium and an inability to convert native vitamin D into its
active state to properly absorb dietary calcium.
Acute symptoms of Hypoparathyroidism are largely due to low
serum calcium and range from muscle pain and tingling, to lack of
focus or ability to concentrate, and anxiety and depression. In
extreme cases, life-threatening events, such as arrhythmias and
seizures, may occur. In the absence of an approved parathyroid
replacement therapy, the standard approach focuses on using large
doses of calcium and active vitamin D to increase calcium levels in
the blood and reduce the severity of symptoms. However, balancing
the administration of large doses of calcium and vitamin D is
challenging due to calcium fluctuations and the long-term use of
this regimen may lead to serious complications. In addition,
calcium and vitamin D do not correct the abnormal bone metabolism
due to PTH deficiency or enable the activation of vitamin D.
About NPS Pharma
NPS Pharma is a global biopharmaceutical company pioneering and
delivering therapies that transform the lives of patients with rare
diseases. The company's current therapeutic areas of focus are rare
gastrointestinal disease and endocrine disorders. These include
Short Bowel Syndrome, a potentially fatal gastrointestinal disorder
in which patients may have to rely on parenteral nutrition for
their survival; Hypoparathyroidism, a complex endocrine disorder in
which the parathyroid glands are either absent or damaged, and the
body produces insufficient or no parathyroid hormone; and Autosomal
Dominant Hypocalcemia, an ultra-rare, genetic disorder of calcium
homeostasis caused by mutations of the calcium-sensing receptor
gene. NPS Pharma continues to seek in-licensing opportunities to
develop new therapies for a broad range of rare diseases, and
complements its proprietary programs with a royalty-based portfolio
of products and product candidates that includes agreements with
Amgen, GlaxoSmithKline, Janssen Pharmaceuticals, and Kyowa Hakko
Kirin. NPS Pharma has operations in the U.S., Canada, Europe, Latin
America and Japan. Learn more at: www.npsp.com.
"NPS Pharma" and "NPS Pharmaceuticals" are the company's
trademarks.
Disclosure notice
Statements made in this press release, which are not historical
in nature, constitute forward-looking statements for purposes of
the safe harbor provided by the Private Securities Litigation
Reform Act of 1995. These statements are based on the company's
current expectations and beliefs and are subject to a number of
factors and uncertainties that could cause actual results to differ
materially from those described in the forward-looking statements.
Forward looking statements include, but are not limited to,
statements concerning the company's future financial performance
and plans for the commercialization of its products, beliefs or
expectations regarding our products in development, statements
concerning the company's plans for international expansion, beliefs
or expectations regarding potential revenue and earnings from
product sales, including beliefs regarding our ability to grow
sales, expectations regarding the market size for our products,
including those in development, and beliefs or expectations
regarding our operating expenses. Risks associated to the company's
business include, but are not limited to, the risks associated with
any failure by the company to successfully commercialize
Gattex/Revestive (teduglutide [rDNA origin]) for injection,
including the risk that physicians and patients may not see the
advantages of Gattex/Revestive and may therefore be reluctant to
utilize the product, the risk that private and public payers may be
reluctant to cover or provide reimbursement for Gattex, risks
related to regulatory approvals for recombinant human parathyroid
hormone 1-84 (rhPTH [1-84]), the risks associated with the
company's strategy, global macroeconomic conditions, the impact of
changes in management or staff levels, the effect of legislation
effecting healthcare reform in the United States, as well as other
risk factors described in the company's periodic filings with the
U.S. Securities and Exchange Commission, including its Annual
Report on Form 10-K and Form 10-Qs. All information in this press
release is as of the date of this press release and NPS undertakes
no duty to update this information, whether as a result of new
information, future events or otherwise.
NPS Pharmaceuticals, Inc.MediaScott Santiamo, +1
908-450-5599SSantiamo@npsp.comorInvestorsSusan Mesco, +1
908-450-5516SMesco@npsp.com
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