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Ascendis Pharma Submits New Drug Application to FDA for Adult Patients with Hypoparathyroidism

• TransCon PTH could, if approved, become the first hormone replacement therapy to address the underlying cause of hypoparathyroidism, an area of major unmet medical need for the estimated 70,000-90,000 adults living with chronic disease in the United States alone.

• European MAA on track for Q4 2022 submission

COPENHAGEN, Denmark, Aug. 31, 2022 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced it has submitted a New Drug Application (NDA) to the U.S. Food & Drug Administration (FDA) 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. TransCon PTH has been granted Orphan designation in the United States and European Union.

“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,” said Dana Pizzuti, Ascendis Pharma’s Senior Vice President and Chief Medical Officer. “Our Phase 2 and Phase 3 trials were the first ever in which most treated patients (86% and 79%, respectively; at 6 months of treatment) achieved normalization of serum calcium and independence from conventional therapy. Further underscoring the potential benefits of our TransCon PTH product candidate, treated patients reported significant reductions in disease-specific physical and cognitive symptoms and significant improvements in their quality of life.”

The NDA is based on data from the global Phase 3 PaTHway Trial and the Phase 2 PaTH Forward Trial, as well as data from the Company’s 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. In these studies, TransCon PTH has been generally well tolerated, with no discontinuations related to study drug.

In the fourth quarter of this year, Ascendis Pharma plans to submit a Marketing Authorisation Application (MAA) for TransCon PTH in hypoparathyroidism to the European Medicines Agency and expects to announce topline results for Pathway Japan, the Phase 3 trial of TransCon PTH in Japan. A phase 3 trial of TransCon PTH in hypoparathyroidism is ongoing in Greater China through VISEN Pharmaceuticals.


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