Efficacy and Safety of TransCon PTH in Adults With Hypoparathyroidism: 52-Week Results From the Phase 3 PaTHway Trial - Summary - MDSpire

Efficacy and Safety of TransCon PTH in Adults With Hypoparathyroidism: 52-Week Results From the Phase 3 PaTHway Trial

  • By

  • Bart L Clarke

  • Aliya A Khan

  • Mishaela R Rubin

  • Peter Schwarz

  • Tamara Vokes

  • Dolores M Shoback

  • Claudia Gagnon

  • Andrea Palermo

  • Lisa G Abbott

  • Lorenz C Hofbauer

  • Lynn Kohlmeier

  • Filomena Cetani

  • Susanne Pihl

  • Xuebei An

  • Alden R Smith

  • Bryant Lai

  • Jenny Ukena

  • Christopher T Sibley

  • Aimee D Shu

  • Lars Rejnmark

  • October 8, 2024

  • 0 min

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Objective:

Assess the long-term efficacy and safety of TransCon PTH (palopegteriparatide) for hypoparathyroidism.

Key Findings:
  • At week 52, 81% of participants met the multicomponent efficacy endpoint.
  • 95% achieved independence from conventional therapy, with none requiring active vitamin D.
  • Sustained improvements in quality of life, physical functioning, and well-being were reported.
  • Mean bone mineral density Z-scores decreased toward age- and sex-matched norms.
  • Mean 24-hour urine calcium excretion decreased from 376 mg/day at baseline to 195 mg/day at week 52.
  • Most treatment-emergent adverse events were mild or moderate, with no trial discontinuations.
Interpretation:

TransCon PTH demonstrated sustained efficacy, safety, and tolerability in adults with hypoparathyroidism over 52 weeks.

Limitations:
  • The study's open-label design may introduce bias.
  • Long-term effects beyond 52 weeks are not yet established.
Conclusion:

TransCon PTH provides a promising alternative to conventional therapy for managing hypoparathyroidism, addressing both biochemical and quality of life outcomes.

Original Source(s)

Efficacy and Safety of TransCon PTH in Adults With Hypoparathyroidism: 52-Week Results From the Phase 3 PaTHway Trial

  • by Bart L Clarke, Aliya A Khan, Mishaela R Rubin, Peter Schwarz, Tamara Vokes, Dolores M Shoback, Claudia Gagnon, Andrea Palermo, Lisa G Abbott, Lorenz C Hofbauer, Lynn Kohlmeier, Filomena Cetani, Susanne Pihl, Xuebei An, Alden R Smith, Bryant Lai, Jenny Ukena, Christopher T Sibley, Aimee D Shu, Lars Rejnmark

  • October 8, 2024

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