FDA Panel Recommends Approval Of NPS Pharmaceuticals, Inc.'s Natpara To Treat Hypoparathyroidism
Published: Sep 15, 2014
September 12, 2014
By Riley McDermid, BioSpace.com Breaking News Sr. Editor
NPS Pharmaceuticals, Inc. has gotten a nod of approval from the a panel at the U.S. Food and Drug Administration for its hormone replacement therapy drug Natpara, the company announced late Friday.
NPS said that the FDA’s Endocrinologic and Metabolic Drugs Advisory Committee voted 8-5 that the available data support the approval of the drug for the long-term treatment of hypoparathyroidism, a rare endocrine disorder characterized by insufficient levels of parathyroid hormone, or PTH.
The FDA isn’t legally bound by the panel’s findings but historically it follows its recommendation. It will make a final decision on the drug by Oct. 24, said NPS.
The company was on a rollercoaster ride in global markets last week, seeing the price of shares rocket 27 percent from close of trading Tuesday, to settle at $31 in after-hours trading Friday. The NASDAQ briefly halted trading of the company’s common stock early Friday as market players waited for the FDA decision.
Natpara is currently designated by the FDA to be an “orphan drug,” or a treatment for a rare disease that affects fewer than 200,000 American patients, and only 180,000 people globally suffer from hypoparathyroidism, according to NPS.
That designated status also will net NPS tax incentives to make Natpara as well as keeping the drug protected by marketing exclusivity clauses for seven years.
Hypoparathyroidism is a rare condition in which the parathyroid glands fail to produce sufficient amounts of PTH or the PTH produced lacks biologic activity. The hormone plays a central role in a variety of critical physiological functions in the body, and insufficient levels of PTH lead to many physiological abnormalities, including low serum calcium and an inability to convert native vitamin D into its active state to properly absorb dietary calcium.
Acute symptoms range from muscle pain and tingling, to lack of focus or ability to concentrate, and anxiety and depression. In extreme cases, life-threatening events, such as arrhythmias and seizures, may occur.
The condition cannot be controlled with calcium and vitamin D in almost half of all cases.