EFFICACY AND SAFETY OF PALOPEGTERIPARATIDE IN CHRONIC HYPOPARATHYROIDISM - A SYSTEMATIC LITERATURE REVIEW
Author(s)
Adalton G. Ribeiro, Sr., Master1, Fernanda Gimenes, Sra, Master2, Celina Migliavaca, Sra, PhD3.
1HEOR, Adium Pharma, São Paulo, Brazil, 2Market Access, Adium Pharma, São Paulo, Brazil, 3HEOR, Inova Medical, Porto Alegre, Brazil.
1HEOR, Adium Pharma, São Paulo, Brazil, 2Market Access, Adium Pharma, São Paulo, Brazil, 3HEOR, Inova Medical, Porto Alegre, Brazil.
OBJECTIVES: To evaluate the efficacy and safety of palopegteriparatide in adults with chronic hypoparathyroidism.
METHODS: A systematic literature review was conducted using a PICOT framework, including adults (≥18 years) with chronic hypoparathyroidism treated with palopegteriparatide versus placebo or conventional therapy (calcium and active vitamin D). Searches were performed in MEDLINE, Embase, Cochrane CENTRAL, and ClinicalTrials.gov (January 2026) without restrictions on language or publication date. Randomized controlled trials (RCTs) and systematic reviews were eligible. Study selection, data extraction, and risk of bias assessment (RoB 2) were conducted by independent reviewers. Certainty of evidence was assessed using the GRADE approach.
RESULTS: A total of 189 records were identified, and two RCTs were included (PaTH Forward [phase II] and PaTHway [phase III]). Palopegteriparatide demonstrated substantial efficacy in achieving normocalcemia and reducing dependence on conventional therapy. In the phase III PaTHway trial, the composite endpoint (normal calcium levels, independence from active vitamin D, and low-dose calcium supplementation) was achieved in 79% of patients receiving palopegteriparatide versus 5% receiving placebo. Independence from active vitamin D (up to 98%) and calcium supplementation (up to 93%) was also observed. Improvements in quality of life were reported. Adverse events were common but mostly mild to moderate, including injection-site reactions, headache, and nausea. Serious adverse events were infrequent and not treatment-related. Both studies had low risk of bias, and the certainty of evidence was high.
CONCLUSIONS: Palopegteriparatide is an effective and well-tolerated treatment, enabling normalization of calcium levels and reducing reliance on conventional therapy in chronic hypoparathyroidism.
METHODS: A systematic literature review was conducted using a PICOT framework, including adults (≥18 years) with chronic hypoparathyroidism treated with palopegteriparatide versus placebo or conventional therapy (calcium and active vitamin D). Searches were performed in MEDLINE, Embase, Cochrane CENTRAL, and ClinicalTrials.gov (January 2026) without restrictions on language or publication date. Randomized controlled trials (RCTs) and systematic reviews were eligible. Study selection, data extraction, and risk of bias assessment (RoB 2) were conducted by independent reviewers. Certainty of evidence was assessed using the GRADE approach.
RESULTS: A total of 189 records were identified, and two RCTs were included (PaTH Forward [phase II] and PaTHway [phase III]). Palopegteriparatide demonstrated substantial efficacy in achieving normocalcemia and reducing dependence on conventional therapy. In the phase III PaTHway trial, the composite endpoint (normal calcium levels, independence from active vitamin D, and low-dose calcium supplementation) was achieved in 79% of patients receiving palopegteriparatide versus 5% receiving placebo. Independence from active vitamin D (up to 98%) and calcium supplementation (up to 93%) was also observed. Improvements in quality of life were reported. Adverse events were common but mostly mild to moderate, including injection-site reactions, headache, and nausea. Serious adverse events were infrequent and not treatment-related. Both studies had low risk of bias, and the certainty of evidence was high.
CONCLUSIONS: Palopegteriparatide is an effective and well-tolerated treatment, enabling normalization of calcium levels and reducing reliance on conventional therapy in chronic hypoparathyroidism.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
CO217
Topic
Clinical Outcomes, Epidemiology & Public Health, Health Technology Assessment
Topic Subcategory
Comparative Effectiveness or Efficacy
Disease
Diabetes/Endocrine/Metabolic Disorders (including obesity), Rare & Orphan Diseases