FDA Committee Recommends NPS's Natpara For Hypoparathyroidism
By Cyndi Root
NPS Pharmaceuticals announced in a press release that a Food and Drug Administration (FDA) committee has voted 8 to 5 to recommend Natpara (rhPTH [1-84]) for the treatment of Hypoparathyroidism. The FDA will consider the Endocrinologic and Metabolic Drugs Advisory Committee’s opinion on Natpara, but is not bound by it, when it acts on the Biologics License Application (BLA) by the Prescription Drug User Fee Act (PDUFA) date of October 24, 2014. Francois Nader, MD, president and CEO of NPS Pharma, said, "As a bioengineered replacement therapy targeting the underlying cause of the disorder, Natpara has been shown in clinical studies to maintain serum calcium while demonstrating similar physiologic effects to the native parathyroid hormone.”
Natpara
Dr. Nader stated that the committee’s positive opinion signifies that Natpara is a long-term treatment for hypoparathyroidism, a rare endocrine disorder characterized by insufficient levels of parathyroid hormone (PTH). Natpara is a bioengineered PTH therapy, granted orphan drug status by the FDA and the European Medicines Agency (EMA) under the trade name Natpar. The FDA committee reviewed several clinical studies, including a Phase 3 study (REPLACE) and a pharmacology study. Interested parties can review the FDA’s Briefing Documents prepared in advance of the committee meeting, which include nonclinical pharmacology/toxicology reports, a clinical summary, a statistical summary, and a pharmacology summary.
Current Hypoparathyroidism Treatments
Hypoparathyroidism is a chronic disorder, usually requiring life-long treatment. Regular blood tests are necessary to determine adequate calcium levels. Patients often take calcium and vitamin D supplements, however side effects from high doses include constipation. Some studies have indicated that treatment with
recombinant human parathyroid hormone (PTH) is beneficial but that treatment is not currently approved. For immediate symptom relief, some patients are admitted to the hospital for intravenous (IV) infusion of calcium. A thiazide diuretic such as hydrochlorothiazide or metolazone is utilized in some cases to increase blood-calcium levels.
About NPS Pharma
NPS Pharma operates worldwide in the U.S., Canada, Europe, Latin America, and Japan. It focuses on rare diseases, gastrointestinal diseases, and endocrine disorders, including Short Bowel Syndrome, Hypoparathyroidism, and Autosomal Dominant Hypocalcemia. The company continues to seek in-licensing opportunities to complement those underway with Amgen, GlaxoSmithKline, Janssen Pharmaceuticals, and Kyowa Hakko Kirin.