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Orphan Drugs: NPS Pharmaceuticals’ Natpara Receives EC Designation

January 3, 2014

NPS Pharmaceuticals, a New Jersey-based biopharmaceutical company developing treatments for rare diseases, announces January 3, 2014, that the European Commission (EC) grants Orphan Drug Designation (ODD) to Natpara (Recombinant Human Parathyroid Hormone (rhPTH[1-84]), for the treatment of Hypoparathyroidism. Natpara is a bioengineered replacement for endogenous Parathyroid Hormone (PTH) – “a hormone replacement therapy for the underlying cause of Hypoparathyroidism”.

Hypoparathyroidism is the decreased function of the parathyroid glands resulting in the under production of PTH. PTH’s functions are :

•   Modulation of serum calcium and phosphate
•   Regulation of renal excretion of phosphate and calcium
•   Activation of Vitamin D
•   Maintenance of normal bone turnover.

Natpara Regulatory Activities

•   In 2007, receives FDA ODD Designation

•   In October 2013, submission of US Biologic License Application (BLA) to FDA

•   In January 2014, receives EC ODD.

NPS Pharmaceuticals also makes orphan drug Gattex, an injectable treatment for rare disease Short Bowel Syndrome (SBS).

References

NPS Pharmaceuticals Pipeline.

Please Note: “Nessler Cylinders” by Panek (Own work) [GFDL or CC-BY-3.0 | via Wikimedia Commons. Copyright © 2012-2014, Orphan Druganaut Blog. All rights reserved.

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