FDA announces priority review to TransCon PTH in hypoparathyroidism
Ascendis Pharma announced that the FDA has accepted for Priority Review its New Drug Application (NDA) for TransCon PTH (palopegteriparatide) in adult patients with hypoparathyroidism and has set a Prescription Drug User Fee Act (PDUFA) target action date of April 30, 2023
TransCon PTH is an investigational prodrug designed to restore parathyroid hormone (PTH [1-34]) to physiological levels over 24 hours in adult patients with hypoparathyroidism. The FDA said that it is not currently planning to hold an advisory committee meeting to discuss the application.
Ascendis plans to submit a Marketing Authorisation Application (MAA) for TransCon PTH in adult patients with hypoparathyroidism to the European Medicines Agency (EMA) during the fourth quarter of this year. During the fourth quarter, the Company also expects to announce topline results for PaTHway Japan, the Phase III trial of TransCon PTH in adult Japanese patients with hypoparathyroidism.
The Phase III PaTHway Trial of TransCon PTH in adults with hypoparathyroidism (HP) demonstrated statistically significant improvement with TransCon PTH compared to control on the primary composite endpoint and all key secondary endpoints. The primary endpoint – defined as serum calcium levels in the normal range (8.3–10.6 mg/dL) and independence from conventional therapy (active vitamin D and >600 mg/day of calcium supplements) with no increase in prescribed study drug within the 4 weeks prior to the Week 26 visit – was achieved by 78.7% of TransCon PTH-treated patients (48 of 61), compared to 4.8% for patients (1 of 21) in control group (p-value <0.0001).
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