Early US Real-World Treatment Patterns and Outcomes in Palopegteriparatide Treatment for Patients with Hypoparathyroidism

To examine the impact of palopegteriparatide (YORVIPATH®; TransCon® PTH) on real-world clinical outcomes in adult patients with hypoparathyroidism. Adult patients with hypoparathyroidism who enrolled in the US expanded access program (EAP) as of October 2024 and provided consent were included in the analysis. Palopegteriparatide was administered at a recommended starting dose of 18 μg/day and titrated alongside conventional therapy (calcitriol and calcium). Conventional therapy requirements, palopegteriparatide dosing, serum calcium levels, and adverse events were assessed up to 12 months of treatment. Among 135 patients enrolled in the EAP who received at least one dose of palopegteriparatide, 123 provided consent for data use and were included in analysis. Nearly all patients (95.1%, 117/123) were previously treated with short-lived PTH therapy (primarily teriparatide or rhPTH (1-84)), and 50.4% (62/123) switched directly from short-lived PTH or PTHrP therapy to palopegteriparatide. There was no clinically meaningful difference (>3 μg) in palopegteriparatide dose between direct switch and non-direct switch patients, with a similar trend over time. With palopegteriparatide treatment, the proportion of patients achieving independence from conventional therapy (defined as taking no calcitriol and ≤600 mg/day of elemental calcium) increased over 12 months. Mean serum calcium levels remained within the reference range (8.3-10.6 mg/dL) with palopegteriparatide, and no new safety signals were identified with up to 12 months of treatment. This real-world analysis of palopegteriparatide treatment of hypoparathyroidism outside of a clinical trial setting reaffirms its efficacy and safety profile and provides insights into outcomes associated with different treatment transition practices.