NPS Pharmaceuticals Observes Rare Disease Day 2013 and Celebrates 30th Anniversary of National Organization for Rare Disorders

  NPS Pharmaceuticals Observes Rare Disease Day 2013 and Celebrates 30th
  Anniversary of National Organization for Rare Disorders

          -- Nearly one in 10 Americans live with a rare disease --

Business Wire

BEDMINSTER, N.J. -- February 28, 2013

NPS Pharmaceuticals, Inc. (NASDAQ: NPSP), a biopharmaceutical company
pioneering and delivering therapies that transform the lives of patients with
rare diseases worldwide, is joining the National Organization for Rare
Disorders (NORD) and other organizations around the world today to observe
World Rare Disease Day. In the U.S., the observance is led by NORD and focuses
on the 25-30 million people living with a rare disease, many of whom have
difficulty getting the right care because of a lack of education about these
disorders.

This year also marks the 30^th anniversaries of NORD and the Orphan Drug Act.
The legislation, which was driven by a determined group of individuals that
also founded NORD, encourages the development and commercialization of
treatments for rare diseases, or “orphan” drugs. The act was designed to
provide financial incentives, including seven-year market exclusivity for an
FDA-approved treatment and an accelerated review process for drug candidates
that qualify. Shortly after the act was signed, NORD was officially formed and
30 years later, the organization is widely recognized as the leading advocate
for awareness and recognition of the challenges faced by people living with
rare diseases.

“Considering about one in every ten people in the U.S. is living with some
form of rare disease and as many as 60 million worldwide, Rare Disease Day is
an observance that truly transcends cultures and borders and is deserving of
international attention,” said Francois Nader, M.D., president and chief
executive officer of NPS Pharmaceuticals. “We at NPS recognize NORD as a noble
champion of patient advocacy in the rare disease community and applaud the
organization for its 30 years of service.”

In the U.S., any disease affecting fewer than 200,000 Americans is considered
rare. According to the National Institutes of Health (NIH), there are
approximately 7,000 such diseases, yet less than 5 percent of these disorders
have FDA-approved therapies.

“While we have made great strides in the last thirty years to raise
international awareness and recognition of rare diseases, we have a renewed
commitment toward our goal of identifying, treating, and ultimately curing
rare disorders through programs encouraging education, advocacy, research and
service,” said Peter L. Saltonstall, president and CEO of NORD. “As we
celebrate this milestone anniversary, NORD is proud to have the support of
organizations such as NPS Pharmaceuticals who are bringing rare disease
treatments to patients in need.”

About Rare Disease Day

Launched in Europe in 2008, World Rare Disease Day was recognized last year in
more than 60 countries, each of which has a national sponsor. NORD is the
sponsor in the U.S.

Each year, Rare Disease Day organizers around the world agree beforehand on a
slogan to express the specific message for that year. For 2013, the slogan is
“Rare Disorders Without Borders” to convey the global solidarity of the rare
disease community. Rare disease patients all over the world face many of the
same medical, social and economic challenges. Together, in solidarity, the
global rare disease community is determined to build a better world for
patients. Visit the Rare Disease Day website
(http://www.rarediseaseday.org/article/rare-disorders-solidarity-has-no-border)
to learn more.

In the U.S., the coalition supporting Rare Disease Day includes patient
organizations and advocacy groups, medical professionals and associations,
government agencies, researchers, and companies developing treatments for rare
diseases. More than 500 patient organizations, government entities, research
institutions, and companies developing treatments have signed up as Rare
Disease Day Partners on the national website hosted by NORD
(www.rarediseaseday.us).

About NORD

The National Organization for Rare Disorders (NORD) represents the nearly 30
million Americans affected by rare diseases. Founded in 1983, NORD is a
non-profit organization providing programs of education, advocacy, research,
and patient services. NORD administers patient assistance programs to help
uninsured or under-insured individuals obtain medications they could not
otherwise afford. It also provides information to patients and their families,
research grants and fellowships, and advocacy on important public policy
issues. Follow NORD at www.rarediseases.org and on Twitter at @RareDiseases.

About Short Bowel Syndrome

Short bowel syndrome (SBS) is a highly disabling condition that can impair a
patient's quality of life and lead to serious life-threatening complications.
SBS typically arises after extensive resection of the bowel due to Crohn's
disease, ischemia or other conditions. SBS patients often suffer from
malnutrition, severe diarrhea, dehydration, fatigue, osteopenia, and weight
loss due to the reduced intestinal capacity to absorb nutrients, water and
electrolytes. The usual treatment for SBS is nutritional support, including
parenteral nutrition (PN) and/or intravenous (IV) fluids to supplement and
stabilize nutritional needs.

Although PN can provide nutritional support for SBS patients, it does not
improve the body's own ability to absorb nutrients. PN is associated with
serious complications, such as infections, blood clots or liver damage, and
the risks increase the longer patients are on PN. Patients on PN often
experience poor quality of life with difficulty sleeping, and frequent
urination, and patients receiving chronic PN often experience a loss of
independence.

About Hypoparathyroidism

Hypoparathyroidism is a rare disorder in which the body produces insufficient
levels of parathyroid hormone, the principal regulator of calcium and
phosphorus. When the body has too little parathyroid hormone, blood calcium
levels drop and phosphorus levels increase, which can cause muscular and
neurological symptoms, as well as bone impairments. There is no approved
hormone replacement for hypoparathyroidism. It is one of the few remaining
hormone deficiency syndromes in which replacement therapy using the native
hormone is not clinically available. Hypoparathyroidism is currently managed
with large doses of calcium supplementation and active vitamin D therapy to
raise the calcium levels in the blood and reduce the severity of symptoms.
Over time, calcium may build up in the body and result in serious health
risks, including calcifications in the kidneys, heart or brain.

About NPS Pharmaceuticals

NPS Pharmaceuticals is a biopharmaceutical company pioneering and delivering
therapies that transform the lives of patients with rare diseases worldwide.
The company’s lead product, Gattex® 0.05 mg/kg/d (teduglutide [rDNA origin])
for injection is FDA-approved for the treatment of adult patients with short
bowel syndrome (SBS) who are dependent on parenteral support. NPS is also
developing Natpara® (rhPTH[1-84]) for the treatment of adult
hypoparathyroidism and expects to submit its Biologic License Application
(BLA) to the FDA in 2013.

NPS's earlier stage pipeline includes two calcilytic compounds, NPSP790 and
NPSP795, with potential application in rare disorders involving increased
calcium receptor activity, such as autosomal dominant hypocalcemia with
hypercalciuria (ADHH). NPS complements its proprietary programs with a
royalty-based portfolio of products and product candidates that includes
agreements with Amgen, GlaxoSmithKline, Janssen Pharmaceuticals, Kyowa Hakko
Kirin, and Takeda GmbH.

"NPS," "NPS Pharmaceuticals," "Gattex," and "Natpara" are the company's
trademarks. All other trademarks, trade names or service marks appearing in
this press release are the property of their respective owners.

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 our
future financial performance. 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, 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, 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.

Contact:

NPS Pharmaceuticals, Inc.
Susan M. Mesco, 908-450-5516
smesco@npsp.com
 
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