Natpara for the treatment of hypoparathyroidism.
BACKGROUND
Hypoparathyroidism is a rare endocrine disorder characterized by hypocalcaemia and hyperphosphatemia, due to absent or inappropriately low parathyroid hormone levels. For management of chronic hypoparathyroidism, current treatment options involve oral calcium and vitamin D. This standard treatment cannot resolve all problematic aspects of the disease, such as abnormal bone remodeling and reduced quality of life, and is associated with long-term complications, including nephrolithiasis, nephrocalcinosis, renal impairment, cataracts and cerebral calcifications.


METHODS
In 2015, the FDA (Food and Drug Administration) approved rhPTH (1-84), named Natpara®, a bioengineered recombinant human PTH, for the management of hypoparathyroidism of any etiology, except Autosomal Dominant Hypocalcemia, not well controlled with calcium and active vitamin D. Herein, the authors review its chemistry, pharmacodynamics, pharmacokinetics and clinical efficacy for hypoparathyroidism.


CONCLUSIONS
Replacement therapy with rhPTH (1-84) is a fundamental step in the treatment of chronic hypoparathyroidism in cases not well controlled by standard therapy, providing the natural hormone that is lacking for the maintenance of normal calcium levels, and reducing long-term risks associated with conventional therapy. Nevertheless, given the chronic nature of the disease, in the future, further studies will have to be performed to evaluate long-term efficacy and safety of the drug.
a Bone Metabolic Diseases Unit, Department of Surgery and Translational Medicine , University of Florence , Florence , Italy.
Gemma
G
Marcucci
b Department of Clinical Medicine and Surgery , Federico II University , Naples , Italy.
Giuseppe
G
Della Pepa
a Bone Metabolic Diseases Unit, Department of Surgery and Translational Medicine , University of Florence , Florence , Italy.
Maria Luisa
ML
Brandi
Hypoparathyroidism; hypocalcemia; parathyroid hormone (PTH); rhPTH (1–84)