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FDA approves Natpara for injection to control hypocalcemia in patients with hypoparathyroidism

NPS Pharmaceuticals, a global biopharmaceutical company pioneering and delivering therapies that transform the lives of patients with rare diseases, announced that the US Food and Drug Administration has approved Natpara (parathyroid hormone) as an adjunct to calcium and vitamin D to control hypocalcemia in patients with hypoparathyroidism.

Hypoparathyroidism is a rare endocrine disorder characterized by insufficient levels of parathyroid hormone, or PTH. Natpara, a bioengineered replica of human PTH, is expected to be available in the second quarter of 2015.

Because of the potential risk of osteosarcoma, Natpara is recommended only for patients who cannot be well-controlled on calcium supplements and active forms of vitamin D alone. Natpara was not studied in patients with hypoparathyroidism caused by calcium-sensing receptor mutations or in patients with acute post-surgical hypoparathyroidism.

"Natpara is a significant advance in the care of hypoparathyroidism and we are very pleased to offer the first and only parathyroid hormone approved for people living with this rare disorder. In clinical studies, Natpara has been shown to increase serum calcium levels and reduce the need for large doses of calcium and active vitamin D," said Francois Nader, MD, president and chief executive officer of NPS Pharma.

"We extend our thanks to the patients and physicians who participated in our clinical development program, as we could not have achieved this important milestone without their contributions."

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

Insufficient levels of PTH lead to low levels of calcium and high levels of phosphate in the blood, and an inability to convert native vitamin D into its active state, which helps the body properly absorb oral calcium.

Parathyroid hormone increases serum calcium by increasing renal tubular calcium reabsorption, increasing intestinal calcium absorption (i.e., by converting native vitamin D (25 OH) into its active form (1,25 OH2 vitamin D)) and by increasing bone turnover which releases calcium into the circulation.

"As someone who has endured the challenges of living with this rare disorder for more than 50 years and seen all five of my children diagnosed with it, I know first-hand how devastating hypoparathyroidism can be and how important it is to have new treatment options," said James Sanders, president of the Hypoparathyroidism Association.

"It’s critical that companies like NPS Pharma continue to research and develop medicines for people with rare diseases, as so many of us are often overlooked."

The FDA approval of Natpara was supported by 12 pharmacology studies and four company-sponsored 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 patients with hypoparathyroidism.

"Patients with hypoparathyroidism may benefit from having a replica of the actual human parathyroid hormone molecule that they are lacking," said Tamara Vokes, MD, professor of medicine at the University of Chicago, and program director of the University of Chicago Fellowship Training Program in Diabetes, Endocrinology and Metabolism.

"In clinical studies, Natpara has been shown to control hypocalcemia in patients with hypoparathyroidism and reduce their need for oral calcium and active vitamin D."

Natpara will be made available through a Risk Evaluation and Mitigation Strategy (REMS) Program to mitigate the potential risk of osteosarcoma.

Natpara contains a Boxed Warning citing the potential risk of osteosarcoma. See below for Important Safety Information about Natpara, including the Boxed Warning, and Warnings and Precautions.

In Europe, the European Medicines Agency (EMA) has validated and initiated its review of NPS Pharma’s marketing authorization application for Natpar.