Ascendis’ TransCon PTH Granted Orphan Drug Status to Treat Hypoparathyroidism

The U.S. Food and Drug Administration has granted orphan drug designation to TransCon PTH, an investigative therapy by Ascendis Pharma to treat hypoparathyroidism.

Hypoparathyroidism is a rare endocrine disorder characterized by insufficient levels of parathyroid hormone (PTH), resulting in low calcium and elevated phosphate levels in the blood.

TransCon PTH is designed to restore parathyroid hormone levels, normalizing blood and urinary calcium levels, serum phosphate levels, and bone turnover.

This technology extends the duration of the therapy and maximizes its action on the body by controlling its release at predefined rates.

“We are pleased about the [orphan drug designation] for TransCon PTH, which reflects the need for a true PTH replacement therapy that more fully addresses all aspects of hypoparathyroidism,” Jonathan Leff, MD, Ascendis Pharma’s chief medical officer, said in a press release.

Recent results from a Phase 1 clinical trial showed that TransCon PTH, when administrated to healthy volunteers, lasted more than 60 hours in the blood circulation, supporting a once-daily administration.

Single ascending doses of TransCon PTH also led to dose-dependent increases of calcium levels in the blood, which were sustained for more than 72 hours. The therapy candidate also demonstrated the expected effects in renal calcium levels.

“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 Leff, of Ascendis. “We are committed to developing innovative new therapies that improve patients’ lives and look forward to advancing TransCon PTH.”

Based on these positive Phase 1 results, Ascendis plans to initiate a Phase 3 program for the treatment early in 2019,”pending discussions with regulatory agencies,” according to the press release.

Orphan drug status aims to encourage companies and researchers to develop therapies for rare and serious diseases through benefits such as seven years of market exclusivity if the product is approved, tax credits for research, and exemption from FDA user fees.