Ascendis Pharma ASND has submitted a New Drug Application (NDA) to the U.S. Food & Drug Administration (FDA) for TransCon PTH, an investigational pro-drug designed to restore parathyroid hormone (PTH [1-34]) to physiological levels over 24 hours in adult patients with hypoparathyroidism.
The regulatory submission is based on data from the global Phase 3 PaTHway Trial and the Phase 2 PaTH Forward Trial, in addition to the data from its ongoing open-label extension studies for both trials. In these studies, TransCon PTH has been generally well tolerated, with no discontinuations related to study drug.
Dana Pizzuti, Senior Vice President and Chief Medical Officer, commented: "We believe the best way to treat hypoparathyroidism is to replace the missing endogenous hormone at stable, physiological levels 24 hours a day. Accordingly, our data support that TransCon PTH, if approved, could become the first-in-class therapy to address the underlying cause of disease and address the urgent needs of patients in the U.S., who today have no such treatment options available.”
TransCon PTH has been granted Orphan designation in the United States and European Union.
The company is planning to submit a Marketing Authorisation Application (MAA) for TransCon PTH in hypoparathyroidism to the European Medicines Agency during the fourth quarter of 2022.
Further, Ascendis expects to announce top-line results from Pathway Phase 3 trial of TransCon PTH in Japan in the fourth quarter.
Price Action : Ascendis shares are trading around 4 percent higher at $89.15 on Wednesday at the time of publication.
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