An investigational hypoparathyroidism treatment, TransCon PTH (Ascendis) has been granted Orphan Drug Designation by the Food and Drug Administration (FDA). 

TransCon PTH is a long-acting prodrug of parathyroid hormone (PTH) designed to restore PTH to physiologic levels, normalizing blood and urinary calcium levels, serum phosphate levels and bone turnover. A Phase 1 study has been completed and the Company has earmarked the first quarter of 2019 to begin a Phase 3 program for the treatment. 

“We have recently completed a Phase 1 trial in healthy adults, which reinforced the potential of TransCon PTH to restore PTH to physiological levels for 24 hours per day,” said Jonathan Leff, MD, and CMO of Ascendis. “We are committed to developing innovative new therapies that improve patients’ lives and look forward to advancing TransCon PTH.”

Hypoparathyroidism affects approximately 80,000 patients in the US.

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