NPS Pharmaceuticals, Inc. (NASDAQ: NPSP), a biopharmaceutical company pioneering and delivering therapies that transform the lives of patients with rare diseases worldwide, today announced that its pivotal Phase 3 study of Natpara® (recombinant human parathyroid hormone (rhPTH[1-84]), known as REPLACE, was published online in The Lancet Diabetes & Endocrinology. Study findings underscore Natpara’s potential as a replacement therapy for endogenous parathyroid hormone (PTH) in hypoparathyroidism, a rare endocrine disorder characterized by insufficient production of PTH, a principal regulator of the body’s mineral homeostasis.
“Managing hypoparathyroidism can be challenging because it is limited to controlling symptoms, which does not address the underlying cause of the disorder and may have deleterious effects on major organs,” said Michael Mannstadt, MD, Massachusetts General Hospital and Harvard Medical School, and first author on the publication. “This is why we are encouraged by the findings from the REPLACE trial that confirm rhPTH (1-84) has the potential to be the first approved replacement therapy that addresses the underlying absence of parathyroid hormone, an approach long used in other classic endocrine disorders.”
When the body is missing PTH, blood calcium levels drop while phosphate levels increase, which can cause a number of physical and mental symptoms, including fatigue, muscle spasms and cramps, tingling, tetany, seizures, brain fog/mental lethargy, anxiety, and depression. Hypoparathyroidism is the only classic endocrine-deficiency disorder without an FDA-approved replacement therapy. It is currently managed with large doses of calcium and active vitamin D to maintain the calcium levels in the blood and reduce the severity of symptoms. Over time, calcium may build up in the body and result in irreversible calcifications in the kidneys, arteries or brain.
REPLACE, a 24-week international phase 3 study of efficacy and safety of daily subcutaneous Natpara in 134 patients with hypoparathyroidism, is the largest randomized, placebo-controlled clinical trial conducted to date in patients with this rare and complex endocrine disorder. Patients were randomized 2:1 to 50µg subcutaneous once daily Natpara or placebo. In the study active vitamin D and oral calcium were progressively reduced, while Natpara could be titrated up from 50 to 75 and then 100 µg. Patients self-administered treatment for 24 weeks and were then followed for four additional weeks after the completion of the treatment phase.
Select the service that is right for you!COMPARE ALL SERVICES
Jim Cramer and Stephanie Link actively manage a real portfolio and reveal their money management tactics while giving advanced notice before every trade.
- $2.5+ million portfolio
- Large-cap and dividend focus
- Intraday trade alerts from Cramer
- Weekly roundups
Access the tool that DOMINATES the Russell 2000 and the S&P 500.
- Buy, hold, or sell recommendations for over 4,300 stocks
- Unlimited research reports on your favorite stocks
- A custom stock screener
- Upgrade/downgrade alerts
Jim Cramer's protege, David Peltier, identifies the best of breed dividend stocks that will pay a reliable AND significant income stream.
- Diversified model portfolio of dividend stocks
- Alerts when market news affect the portfolio
- Bi-weekly updates with exact steps to take - BUY, HOLD, SELL
All of Real Money, plus 15 more of Wall Street's sharpest minds delivering actionable trading ideas, a comprehensive look at the market, and fundamental and technical analysis.
- Real Money + Doug Kass Plus 15 more Wall Street Pros
- Intraday commentary & news
- Ultra-actionable trading ideas
Our options trading pros provide daily market commentary and over 100 monthly option trading ideas and strategies to help you become a well-seasoned trader.
- 100+ monthly options trading ideas
- Actionable options commentary & news
- Real-time trading community
- Options TV