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

Objective: To examine the impact of palopegteriparatide (YORVIPATH®; TransCon® PTH) on real-world clinical outcomes in adult patients with hypoparathyroidism.

Methods: 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.

Results: 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.

Conclusions: 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.