FDA Panel Votes In Favor Of NPS Pharm's Natpara For Hypoparathyroidism Treatment

(RTTNews) - NPS Pharmaceuticals Inc. (NPSP) announced that the U.S. Food and Drug Administration's or FDA Endocrinologic and Metabolic Drugs Advisory Committee voted unanimously 8 to 5 that the available data support the approval of Natpara (rhPTH [1-84]) for the long-term treatment of Hypoparathyroidism, a rare endocrine disorder characterized by insufficient levels of parathyroid hormone, or PTH.

The company said that the Committee's recommendation will be considered by the FDA in its review of the company's Biologics License Application or BLA for Natpara, which has a Prescription Drug User Fee Act or PDUFA date of October 24, 2014.

The Committee reviewed data from the Natpara clinical development program for Hypoparathyroidism, consisting of one pharmacology study and four efficacy and safety studies. The pivotal Phase 3 study, known as REPLACE, was a randomized, double-blind, placebo controlled study and the largest clinical trial conducted to date in Hypoparathyroidism.

Natpara is a bioengineered replacement therapy for endogenous PTH that NPS Pharma has developed for the treatment of Hypoparathyroidism. The FDA has granted orphan drug status for Natpara for the treatment of Hypoparathyroidism. The European Medicines Agency has also granted orphan drug status for the drug under the trade name Natpar.

Hypoparathyroidism is a rare condition in which the parathyroid glands fail to produce sufficient amounts of parathyroid hormone (PTH) or where PTH lacks biologic activity. PTH plays a central role in a variety of critical physiological functions in the body. In patients with Hypoparathyroidism, 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.