First FDA-approved parathyroid hormone for
hypoparathyroidism
Supported by largest clinical program ever
conducted in patients with hypoparathyroidism
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 U.S. Food and Drug Administration (FDA) has approved
Natpara® (parathyroid hormone) as an adjunct to calcium and vitamin
D to control hypocalcemia in patients with hypoparathyroidism.
Hypoparathyroidism is a rare endocrine disorder characterized by
insufficient levels of parathyroid hormone, or PTH. Natpara, a
bioengineered replica of human PTH, is expected to be available in
the second quarter of 2015.
Because of the potential risk of osteosarcoma, Natpara is
recommended only for patients who cannot be well-controlled on
calcium supplements and active forms of vitamin D alone. Natpara
was not studied in patients with hypoparathyroidism caused by
calcium-sensing receptor mutations or in patients with acute
post-surgical hypoparathyroidism.
“Natpara is a significant advance in the care of
hypoparathyroidism and we are very pleased to offer the first and
only parathyroid hormone approved for people living with this rare
disorder. In clinical studies, Natpara has been shown to increase
serum calcium levels and reduce the need for large doses of calcium
and active vitamin D,” said Francois Nader, MD, president and chief
executive officer of NPS Pharma. “We extend our thanks to the
patients and physicians who participated in our clinical
development program, as we could not have achieved this important
milestone without their contributions.”
Hypoparathyroidism is a rare endocrine disorder in which the
parathyroid glands fail to produce sufficient amounts of
parathyroid hormone (PTH) or where the hormone lacks biologic
activity. PTH plays a central role in a variety of critical
physiological functions in the body. Insufficient levels of PTH
lead to low levels of calcium and high levels of phosphate in the
blood, and an inability to convert native vitamin D into its active
state, which helps the body properly absorb oral calcium.
Parathyroid hormone increases serum calcium by increasing renal
tubular calcium reabsorption, increasing intestinal calcium
absorption (i.e., by converting native vitamin D (25 OH) into its
active form (1,25 OH2 vitamin D)) and by increasing bone turnover
which releases calcium into the circulation.
“As someone who has endured the challenges of living with this
rare disorder for more than 50 years and seen all five of my
children diagnosed with it, I know first-hand how devastating
hypoparathyroidism can be and how important it is to have new
treatment options,” said James Sanders, president of the
Hypoparathyroidism Association. “It’s critical that companies like
NPS Pharma continue to research and develop medicines for people
with rare diseases, as so many of us are often overlooked.”
The FDA approval of Natpara was supported by 12 pharmacology
studies and four company-sponsored efficacy and safety studies. The
pivotal Phase 3 study, known as REPLACE, was a randomized,
double-blind, placebo-controlled study and the largest clinical
trial conducted to date in patients with hypoparathyroidism.
“Patients with hypoparathyroidism may benefit from having a
replica of the actual human parathyroid hormone molecule that they
are lacking,” said Tamara Vokes, MD, professor of medicine at the
University of Chicago, and program director of the University of
Chicago Fellowship Training Program in Diabetes, Endocrinology and
Metabolism. “In clinical studies, Natpara has been shown to control
hypocalcemia in patients with hypoparathyroidism and reduce their
need for oral calcium and active vitamin D.”
Natpara will be made available through a Risk Evaluation and
Mitigation Strategy (REMS) Program to mitigate the potential risk
of osteosarcoma.
Natpara contains a Boxed Warning citing the potential risk of
osteosarcoma. See below for Important Safety Information about
Natpara, including the Boxed Warning, and Warnings and
Precautions.
In Europe, the European Medicines Agency (EMA) has validated and
initiated its review of NPS Pharma’s marketing authorization
application for Natpar™.
NPS Advantage™
NPS Pharma is committed to ensuring that patients have access to
our medicines. To assist patients and healthcare professionals in
facilitating care with Natpara, NPS Pharma has a free support
program called NPS Advantage that includes a dedicated team of care
coordinators and specialized nurses for patients treated with
Natpara. This program is designed to help patients navigate all
aspects of care and includes help with insurance authorizations and
appeals, answering questions about Natpara and its use, and
locating resources for patients to connect them to care. For more
information, please visit www.npsadvantage.com.
NPS Pharma also provides support to independent non-profit
organizations that provide assistance to patients who need help
covering out-of-pocket medication costs.
About Natpara® (parathyroid hormone) for
Injection
Natpara® (parathyroid hormone) for injection is indicated as an
adjunct to calcium and vitamin D to control hypocalcemia in
patients with hypoparathyroidism. Natpara is a bioengineered
replica of human parathyroid hormone.
Because of the potential risk of osteosarcoma, Natpara is
recommended only for patients who cannot be well-controlled on
calcium supplements and active forms of vitamin D alone. Natpara
was not studied in patients with hypoparathyroidism caused by
calcium-sensing receptor mutations or in patients with acute
post-surgical hypoparathyroidism.
In clinical studies, Natpara has been shown to increase serum
calcium levels while reducing the need for oral calcium and active
vitamin D and, in some cases, eliminate the need for active vitamin
D altogether. The most common adverse reactions associated with
Natpara and occurring in greater than 10% of individuals were:
paresthesia, hypocalcemia, headache, hypercalcemia, nausea, and
hypoesthesia, diarrhea, vomiting, arthralgia, hypercalciuria and
pain in extremity.
Natpara is self-administered once daily by subcutaneous
injection. The starting dose of Natpara is 50 mcg once daily.
Natpara received orphan drug status for the treatment of
hypoparathyroidism from the FDA in 2007 and the EMA in 2013.
Important Safety Information
What is NATPARA?
- NATPARA is a prescription parathyroid
hormone (PTH) used with calcium and vitamin D to control low blood
calcium (hypocalcemia) in people with low PTH blood levels
(hypoparathyroidism).
- NATPARA is only for people who do not
respond well to treatment with calcium and active forms of vitamin
D alone, because it may increase the possible risk of bone cancer
(osteosarcoma).
- NATPARA was not studied in people with
hypoparathyroidism caused by calcium sensing receptor
mutations.
- NATPARA was not studied in people who
get sudden hypoparathyroidism after surgery.
- It is not known if NATPARA is safe and
effective for children 18 years of age and younger. NATPARA should
not be used in children and young adults whose bones are still
growing.
What is the most important information I should know about
NATPARA?
NATPARA may cause serious side effects, including:
Possible bone cancer (osteosarcoma).
- During animal drug testing, NATPARA
caused some rats to develop a bone cancer called osteosarcoma. It
is not known if people who take NATPARA will have a higher chance
of getting osteosarcoma.
- NATPARA is only available through
the NATPARA Risk Evaluation and Mitigation Strategy (REMS)
Program. The purpose of the NATPARA REMS program is to inform
patients about the potential risk of osteosarcoma associated with
the use of NAPTARA. For more information about this REMS program,
call 1-855-628-7272 or go to www.NATPARAREMS.com.
High blood calcium (hypercalcemia)
- NATPARA can cause some people to have a
higher blood calcium level than normal.
- Your doctor should check your blood
calcium before you start and during your treatment with
NATPARA
- Tell your doctor if you have nausea,
vomiting, constipation, low energy, or muscle weakness. These may
be signs that you have too much calcium in your blood.
Low blood calcium (hypocalcemia)
- People who stop using or miss a dose of
NATPARA may have an increased risk of severe low blood calcium
levels
- Tell your doctor if you have tingling
of your lips, tongue, fingers and feet, twitching of face muscles,
cramping of feet and hands, seizures, depression, or have problems
thinking or remembering.
Tell your doctor right away if you have any of these signs and
symptoms of high or low blood calcium levels.
Tell your doctor about all the medicines you take,
including prescription and over-the-counter medicines, vitamins,
and herbal supplements.
What are the most common side effects of NATPARA?
The most common side effects of NATPARA include
- Tingling, tickling, or a burning
feeling of your skin (paresthesia), headache and nausea
These are not all the possible side effects of NATPARA. For more
information, ask your doctor. Call your doctor for medical advice
about side effects.
You may report side effects to the NPS Adverse Event/Product
Complaint Line at 1-855-215-5550 or by calling the Food and
Drug Administration (FDA) at 1-800-FDA- 1088 or
www.fda.gov/medwatch.
U.S. full prescribing information for Natpara, including boxed
warning, is available at
http://www.npsp.com/file_depot/0-10000000/0-10000/262/folder/2023/NatparaPI.pdf
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
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 and
Natpara (parathyroid hormone) for injection, including the risk
that physicians and patients may not see the advantages of
Gattex/Revestive or Natpara and may therefore be reluctant to
utilize the products, the risk that private and public payers may
be reluctant to cover or provide reimbursement for Gattex or
Natpara, 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 the company undertakes no duty to update
this information, whether as a result of new information, future
events or otherwise.
NPS Pharmaceuticals, Inc.Media:Scott Santiamo, +1 908
450-5599SSantiamo@npsp.comorInvestors:Susan Mesco, +1 908
450-5516SMesco@npsp.com
Nps (NASDAQ:NPSP)
Historical Stock Chart
From Sep 2024 to Oct 2024
Nps (NASDAQ:NPSP)
Historical Stock Chart
From Oct 2023 to Oct 2024