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Ascendis Pharma's hormone disorder drug gets orphan designation from US FDA

Ascendis Pharma A/S said the U.S. Food and Drug Administration granted orphan drug designation to TransCon PTH for treating hypoparathyroidism, a disorder that results in low levels of the parathyroid hormone.

Low levels of parathyroid hormone can lead to low calcium and elevated phosphate levels in the blood. Over time, the disorder can lead to major complications, such as calcium depositions on soft tissues including the brain, the lens of the eye, and kidneys, resulting in kidney failure.

TransCon PTH is a prodrug, a compound that is metabolized into a pharmacologically active drug after administration.

In a phase 1 study, TransCon PTH has shown the potential to restore the levels of parathyroid hormone, or PTH, to required levels for 24 hours per day.

The drug showed a longer half-life, a measure of drug's ability to remain available in the blood, than current short-acting PTH molecules, such as Shire PLC's Natpara and Eli Lilly and Co.'s Forteo.

Hellerup, Denmark-based Ascendis is planning to launch a phase 3 program for TransCon PTH in the first quarter of 2019, pending discussions with regulatory agencies.

Hypoparathyroidism affects about 80,000 patients in the U.S., the majority of whom develop the condition after damage or accidental removal of the parathyroid glands during thyroid surgery, according to Ascendis.