Early U.S. 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 U.S. expanded access program 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 expanded access program who received at least 1 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 parathyroid hormone (PTH) therapy (primarily teriparatide or recombinant human PTH [1-84]), and 50.4% (62/123) switched directly from short-lived PTH or PTH-related protein therapy to palopegteriparatide. There was no clinically meaningful difference (>3 μg) in palopegteriparatide dose between direct switch and nondirect 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.

CONCLUSION

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.