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
Commission has granted orphan drug designation to Natpara® (recombinant
human parathyroid hormone (rhPTH[1-84]) for the treatment of
hypoparathyroidism. Natpara is a bioengineered replacement for
endogenous parathyroid hormone (PTH) that NPS has developed for the
treatment of hypoparathyroidism, a rare endocrine disorder in which the
body produces insufficient levels of parathyroid hormone, a principal
regulatory of the body's mineral homeostasis. Orphan drug designation in
the European Union (EU) is given to products that are designed for the
diagnosis, prevention or treatment of rare diseases that are
life-threatening or chronically debilitating. A disease is defined as
rare in the EU if it affects no more than five in 10,000 people.
"We are very pleased that Natpara has received orphan drug status in the
EU," said Francois Nader, MD, president and chief executive officer of
NPS Pharmaceuticals. "As a global rare disease biopharmaceutical
company, this aligns with our commitment to deliver innovative therapies
that transform the lives of patients around the world.
Hypoparathyroidism patients face a significant burden of disease given
the multitude of physical, cognitive, and emotional symptoms associated
with this disorder. Natpara could be the first PTH replacement therapy
to treat this condition."
The company was also granted orphan drug status by the U.S. Food and
Drug Administration (FDA) in 2007. The company submitted its U.S.
Biologic License Application to FDA in October 2013.
PTH plays a central role in a variety of critical physiological
functions, including closely modulating serum calcium and phosphate,
regulating renal excretion of calcium and phosphate, activating vitamin
D, and maintaining normal bone turnover. In patients with
hypoparathyroidism, insufficient levels of PTH lead to low serum
calcium, high serum phosphate, increased urinary calcium excretion, and
decreased urinary phosphorus excretion. PTH deficiency can also disrupt
skeletal homeostasis, leading to bone abnormalities. In addition,
patients with insufficient levels of PTH are unable to convert native
vitamin D into itsactive state to properly absorb dietary calcium.
Acute symptoms of hypoparathyroidism are largely due to hypocalcemia and
include fatigue, muscle spasms and cramps, tingling, tetany, seizures,
brain fog/mental lethargy, anxiety, and depression. In the absence of an
approved parathyroid replacement therapy, the standard approach focuses
on using high doses of calcium and active vitamin D to increase calcium
levels in the blood and reduce the severity of hypocalcemic symptoms.
However, balancing the administration of supplements is challenging due
to calcium fluctuations and the long-term use of high doses of calcium
and vitamin D may lead to serious complications, including long-term
renal damage. In addition, because serum phosphate levels are elevated
when PTH is missing, increasing serum calcium may lead to irreversible
calcium-phosphate deposits in the kidneys, arteries or brain. Further,
supplements do not correct the abnormal bone metabolism due to PTH
deficiency or enable the activation of vitamin D.
About NPS Pharmaceuticals
NPS Pharmaceuticals is a global biopharmaceutical company pioneering and
delivering therapies that transform the lives of patients with rare
diseases worldwide. The company's lead product, Gattex® (teduglutide
[rDNA origin]) for injection is approved in the US for adult patients
with Short Bowel Syndrome (SBS) who are dependent on parenteral support.
In the EU, teduglutide (trade name: Revestive®) is approved for the
treatment of adult patients with SBS; patients should be stable
following a period of intestinal adaptation after surgery. Teduglutide
is not approved for the treatment of pediatric SBS patients. The safety
and efficacy of teduglutide in this population is currently being
evaluated in a global registration trial.
NPS has also developed Natpara® (rhPTH [1-84]) for the treatment of
hypoparathyroidism and submitted a Biologics License Application to the
US Food and Drug Administration in October 2013.
NPS' earlier stage pipeline includes NPSP795, a calcilytic compound with
potential application in rare disorders involving increased calcium
sensing receptor activity, such as autosomal dominant hypocalcemia
(ADH). NPS 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. Additional information about NPS is available through its
corporate website, http://www.npsp.com.
"NPS," "NPS Pharmaceuticals," "Gattex," "Revestive," and "Natpara" are
the company's trademarks.
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. 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 (teduglutide [rDNA origin]) for injection,
including the risk that physicians and patients may not see the
advantages of Gattex 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 Natpara (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 release and NPS
undertakes no duty to update this information, whether as a result of
new information, future events or otherwise.
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