Ascendis NDA for Hypoparathyroidism Therapy

Ascendis Pharma has submitted an NDA for TransCon PTH, an investigational prodrug designed to restore parathyroid hormone (PTH [1-34]) to physiological levels over 24 hours in adult patients with hypoparathyroidism. The submission is based on data from the global Phase 3 PaTHway Trial, the Phase 2 PaTH Forward Trial, and an ongoing open-label extension studies for both trials. “Notably, 57 out of 59 patients continue in the open-label portion of the Phase 2 trial beyond two years of treatment, and 78 out of 79 patients continue in the open-label portion of the Phase 3 trial of TransCon PTH,” the company says. “In these studies, TransCon PTH has been generally well tolerated, with no discontinuations related to study drug.”

Hypoparathyroidism is a rare endocrine disorder where patients experience insufficient levels of parathyroid hormone, which results in low calcium and elevated phosphate levels in the blood.