US-based biopharmaceutical company NPS Pharmaceuticals has announced that it has received orphan drug designation from the European Commission for its Natpara (recombinant human parathyroid hormone (rhPTH[1-84]), indicated for the treatment of hypoparathyroidism.
NPS Pharmaceuticals has developed Natpara as a bioengineered replacement for endogenous PTH for the treatment of hypoparathyroidism, an endocrine disorder that is characterised by insufficient levels of parathyroid hormone.
NPS Pharmaceuticals president and CEO Dr Francois Nader said: "As a global rare disease biopharmaceutical company, this aligns with our commitment to deliver innovative therapies that transform the lives of patients around the world.
"Hypoparathyroidism patients face a significant burden of disease given the multitude of physical, cognitive, and emotional symptoms associated with this disorder. Natpara could be the first PTH replacement therapy to treat this condition."
In October 2013, the company presented findings from its pivotal Phase III study of Natpara. The REPLACE study has demonstrated that the investigational therapy has a beneficial effect on bone health in patients with hypoparathyroidism.
The REPLACE study has met the primary efficacy endpoint, which was defined as a 50% or greater reduction in oral calcium supplementation and active vitamin D therapy and a total serum calcium concentration that was normalized or maintained compared with baseline after 24 weeks of treatment.
NPS Pharmaceuticals has also submitted its biologics license application to the US Food and Drug Administration in October 2013.
Insufficient levels of PTH in hypoparathyroidism patients lead to low serum calcium, high serum phosphate, increased urinary calcium excretion, and decreased urinary phosphorus excretion.
The insufficient levels of PTH can also disrupt skeletal homeostasis, which leads to bone abnormalities. In addition, patients with PTH deficiency are unable to convert native vitamin D into its active state to properly absorb dietary calcium.