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 European Commission has granted orphan drug designation to Natpara® (recombinant human parathyroid hormone (rhPTH[1-84]) for the treatment of hypoparathyroidism. Natpara is a bioengineered replacement for endogenous parathyroid hormone (PTH) that NPS has developed for the treatment of hypoparathyroidism, a rare endocrine disorder in which the body produces insufficient levels of parathyroid hormone, a principal regulatory of the body’s mineral homeostasis. Orphan drug designation in the European Union (EU) is given to products that are designed for the diagnosis, prevention or treatment of rare diseases that are life-threatening or chronically debilitating. A disease is defined as rare in the EU if it affects no more than five in 10,000 people.
“We are very pleased that Natpara has received orphan drug status in the EU,” said Francois Nader, MD, president and chief executive officer of NPS Pharmaceuticals. “As a global rare disease biopharmaceutical company, this aligns with our commitment to deliver innovative therapies that transform the lives of patients around the world. Hypoparathyroidism patients face a significant burden of disease given the multitude of physical, cognitive, and emotional symptoms associated with this disorder. Natpara could be the first PTH replacement therapy to treat this condition.”
The company was also granted orphan drug status by the U.S. Food and Drug Administration (FDA) in 2007. The company submitted its U.S. Biologic License Application to FDA in October 2013.
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 vitamin D into its active state to properly absorb dietary calcium.