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The Food and Drug Administration (FDA) has accepted for Priority Review the New Drug Application (NDA) for TransCon™ PTH (palopegteriparatide) in adult patients with hypoparathyroidism.
TransCon PTH is an investigational once-daily long-acting prodrug of parathyroid hormone (PTH[1-34]) designed to restore physiologic levels of PTH for 24 hours each day. The NDA is supported by data from the phase 3 PaTHway trial (ClinicalTrials.gov Identifier: NCT04701203) and the phase 2 PaTH Forward trial (ClinicalTrials.gov Identifier: NCT04009291), which evaluated the efficacy and safety of TransCon PTH in adults with hypoparathyroidism. The application also included data from the open-label extension studies for both trials.
Findings from the PaTHway trial showed that a statistically significantly greater proportion of patients treated with TransCon PTH met the primary endpoint achieving serum calcium levels within normal range and independence from therapeutic levels of conventional therapy compared with placebo. Treatment with TransCon PTH also led to statistically significant improvements in physical and cognitive symptoms.
In the PaTH Forward trial, long-term treatment with TransCon PTH provided a durable response, with 93% of patients achieving independence from conventional therapy with active vitamin D and therapeutic levels of calcium through week 110. Additionally, continued restoration of skeletal bone mineral density (BMD) toward sex- and age-expected norms was observed in TransCon PTH-treated patients.
“We believe results from our clinical trials point to the importance of TransCon PTH as a potential new hormone replacement treatment for adult patients with hypoparathyroidism across disease etiologies studied in our trials,” said Birgitte Volck, Senior Vice President, Head of Clinical Development & Medical Affairs, Endocrinology Rare Diseases, at Ascendis Pharma. “We understand the urgency and need these patients face and will continue our work to make TransCon PTH available as quickly as possible.”
A Prescription Drug User Fee Act target date of April 30, 2023 has been set for the application.
References
- FDA accepts for Priority Review Ascendis Pharma’s NDA for TransCon™ PTH in adult patients with hypoparathyroidism. Copenhagen, Denmark. News release. Ascendis Pharma. October 31, 2022. Accessed November 1, 2022. https://www.globenewswire.com/news-release/2022/10/31/2544511/0/en/FDA-Accepts-for-Priority-Review-Ascendis-Pharma-s-NDA-for-TransCon-PTH-in-Adult-Patients-with-Hypoparathyroidism.html
- New long-term data in adult patients with hypoparathyroidism treated with Ascendis Pharma’s TransCon™ PTH through week 110 in the PaTH Forward trial presented at ASBMR 2022. Copenhagen, Denmark. News release. Ascendis Pharma. September 12, 2022. Accessed November 1, 2022. https://investors.ascendispharma.com/news-releases/news-release-details/new-long-term-data-adult-patients-hypoparathyroidism-treated
