NPS Pharmaceuticals, Inc. (NASDAQ:NPSP), a global biopharmaceutical
company pioneering and delivering therapies that transform the lives of
patients with rare diseases worldwide, today announced that the U.S.
Food and Drug Administration (FDA) has accepted and filed for review the
company's Biologics License Application (BLA) for Natpara® (recombinant
human parathyroid hormone 1-84, (rhPTH[1-84])) for the treatment of
hypoparathyroidism. Under the Prescription Drug User Fee Act (PDUFA),
the goal date for a decision by the FDA is October 24, 2014.
Natpara is a bioengineered replacement for endogenous parathyroid
hormone (PTH) that NPS has developed for the treatment of
hypoparathyroidism, a rare endocrine disorder characterized by
insufficient levels of parathyroid hormone, a principal regulator of the
body's mineral homeostasis.
"We are pleased that the FDA accepted our BLA filing for Natpara and are
looking forward to continuing our collaboration with the Agency as we
move towards delivering the first replacement therapy to patients with
hypoparathyroidism," said Roger Garceau, MD, FAAP, executive vice
president and chief medical officer of NPS Pharmaceuticals.
The company's clinical development program for Natpara includes 12
pharmacology studies, five efficacy and safety studies in
hypoparathyroidism, and a supporting development program consisting of
seven studies in osteoporosis. The pivotal Phase 3 study known as
REPLACE, was a randomized, double-blind, placebo controlled study of 134
patients with hypoparathyroidism.
The FDA and European Medicines Agency have granted orphan drug status
for Natpara for the treatment of hypoparathyroidism.
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
vitmin D into its active 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," "Natpara," "Preotact," and
"Revestive" 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 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 (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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