NPS Pharmaceuticals, Inc. (NASDAQ:NPSP), a global biopharmaceutical company pioneering and delivering therapies that transform the lives of patients with rare diseases worldwide, today announced that the U.S. Food and Drug Administration (FDA) has accepted and filed for review the company’s Biologics License Application (BLA) for Natpara ® (recombinant human parathyroid hormone 1-84, (rhPTH[1-84])) for the treatment of hypoparathyroidism. Under the Prescription Drug User Fee Act (PDUFA), the goal date for a decision by the FDA is October 24, 2014.
Natpara is a bioengineered replacement for endogenous parathyroid hormone (PTH) that NPS has developed for the treatment of hypoparathyroidism, a rare endocrine disorder characterized by insufficient levels of parathyroid hormone, a principal regulator of the body’s mineral homeostasis.
“We are pleased that the FDA accepted our BLA filing for Natpara and are looking forward to continuing our collaboration with the Agency as we move towards delivering the first replacement therapy to patients with hypoparathyroidism,” said Roger Garceau, MD, FAAP, executive vice president and chief medical officer of NPS Pharmaceuticals.
The company’s clinical development program for Natpara includes 12 pharmacology studies, five efficacy and safety studies in hypoparathyroidism, and a supporting development program consisting of seven studies in osteoporosis. The pivotal Phase 3 study known as REPLACE, was a randomized, double-blind, placebo controlled study of 134 patients with hypoparathyroidism.The FDA and European Medicines Agency have granted orphan drug status for Natpara for the treatment of hypoparathyroidism. About Hypoparathyroidism 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.