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. 2025 Aug 20:S1530-891X(25)01003-1.
doi: 10.1016/j.eprac.2025.08.008. Online ahead of print.

Early U.S. Real-World Treatment Patterns and Outcomes in Palopegteriparatide Treatment for Patients With Hypoparathyroidism

Timothy Graham  1 , Dolores M Shoback  2 , Lisa Abbott  3 , Sara Lubitz  4 , Gary Edelson  5 , Adnan Haider  6 , Steven W Ing  7 , Micol S Rothman  8 , Natalie E Cusano  9 , Carol Zhao  10 , Jenny Ukena  10 , Mark Schneider  10 , Christopher T Sibley  11 , Mishaela R Rubin  12
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Free article

Early U.S. Real-World Treatment Patterns and Outcomes in Palopegteriparatide Treatment for Patients With Hypoparathyroidism

Timothy Graham et al. Endocr Pract. .
Free article

Abstract

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.

Keywords: TransCon PTH; hypoparathyroidism; palopegteriparatide; real-world data; treatment switching.

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Conflict of interest statement

Disclosure Carol Zhao, Jenny Ukena, Mark Schneider, and Christopher T. Sibley are all full-time employees of Ascendis Pharma. Dolores M. Shoback received research and salary funding from Bone Health Tech and research funding from Ascendis Pharma. Lisa Abbott is a research investigator for Ascendis Pharma and Takeda and a speaker for AbbVie and Clarus. Natalie E. Cusano is a speaker for Alexion, a consultant for Ascendis Pharma, and a researcher for Shire Pharmaceuticals/Takeda. Mishaela R. Rubin is a consultant for MBX Biosciences, receives honoraria from Ascendis Pharma, and serves on the speaker’s bureau for Ascendis Pharma. Timothy Graham, Sara Lubitz, Adnan Haider, Steven W. Ing, Michol S. Rothman, and Gary Edelson report no interests to disclose at this time.

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