The European Commission has granted Bedminster company NPS Pharmaceuticals Inc. orphan drug status for Natpara, a hormone replacement drug being developed to treat adult hypoparathyroidism, a rare endocrine disorder that causes blood calcium levels to fall.
The approval by the European body, similar to one NPS obtained from the U.S. Food and Drug Administration, provides incentives to encourage breakthrough treatments for diseases that afflict small populations, defined by the European Commission as no more than 5 in 10,000 people.
NPS envisions a global market for treating hypoparathyroidism, a disease the company estimates affects 80,000 Americans as well. NPS submitted a marketing application to the FDA last year.
Hypoparathyroidism is a disorder in which the parathyroid glands in the neck do not produce enough parathyroid hormone necessary to regulate calcium and phosphorous levels. Symptoms can include muscle cramps, spasms, weakness, fatigue, memory loss and depression.
"Hypoparathyroidism patients face a significant burden of disease given the multitude of physical, cognitive, and emotional symptoms associated with this disorder," NPS CEO Francois Nader said in a statement Friday. "Natpara could be the first parathyroid hormone replacement therapy to treat this condition."
The FDA granted orphan drug status for Natpara in 2007. The designation provides incentives such as grants and exemption from regulatory fees. If the drug is approved, the status can also provide market exclusivity arrangements and tax credits on future profits.
Shares of NPS rose 7 cents to $31.78 in morning trading on Nasdaq. Its stock has more than tripled in the past 12 months.
NPS' lead product is Gattex, which treats patients suffering from short bowel syndrome, a rare disorder that prevents the body from properly absorbing nutrients. The company reports about $128 million in annual revenue.