Aliya Aziz Khan, MD, Clinical Professor of Medicine McMaster University and Director of the Calcium Disorder Clinic, discusses long-term safety and efficacy results of palopegteriparatide in patients with hypoparathyroidism.
Hypoparathyroidism is an endocrine disorder in which the parathyroid glands in the neck do not produce enough parathyroid hormone (PTH). Common signs and symptoms include abdominal pain, brittle nails, cataracts, dry hair and skin, muscle cramps, tetany, pain in the face, legs, and feet, seizures, tingling sensation, and weakened tooth enamel. It may be caused by injury to the parathyroid glands (e.g., during surgery).
Data was recently presented at the ENDO 2025 meeting on the evaluation of the long-term safety and efficacy of palopegteriparatide in adults with chronic hypoparathyroidism through week 156 of the PaTHway trial. Palopegteriparatide is a prodrug of PTH designed to provide active PTH within the physiological range for 24 hours a day. It is administered subcutaneously once daily and is approved by the U.S. Food and Drug Administration (FDA)
The PaTHway study was a phase 3, randomized, double-blind, placebo-controlled, 26-week trial with an ongoing open-label extension period. At week 156, 89% of participants remained in the trial and of those, 96% were independent from conventional therapy of vitamin D and calcium. Additionally, 88% of participants had normal albumin-adjusted serum calcium levels with a mean of 8.9 mg/dL and mean serum phosphate can calcium x phosphate product levels remained within normal ranges through week 156.
Mean estimated glomerular filtration rate (eGFR) was 78.0 mL/min/1.73 m2 at week 156, highlighting a mean increase of 8.8 mL/min/1.73 m2 from baseline. 59% and 43% of participants had an increase in eGFR of ≥5 mL/min/1.73 m2 and ≥10 mL/min/1.73 m2, respectively. Among those with baseline eGFR <60 mL/min/1.73 m2, the mean increase in eGFR was 14.0 mL/min/1.73 m2 from baseline to week 156. Mean 24-hour urine calcium levels normalized with palopegteriparatide, remaining below the upper limit of normal through week 156.
Treatment-emergent adverse events were mostly grades 1 or 2, with no new safety signals observed.
To learn more about hypoparathyroidism and other rare endocrine disorders, visit https://checkrare.com/diseases/endocrine-disorders/