The U. S. Food and Drug Administration (FDA) has approved palopegteriparatide (Yorvipath) for the treatment of hypoparathyroidism in adults, the drug’s manufacturer, Ascendis Pharma, announced.
In hypoparathyroidism, inadequate levels of parathyroid hormone (PTH) can lead to hypocalcemia and hyperphosphatemia, and subsequent headaches add neuromuscular irritability and renal and vascular headaches. Cognitive disorders are also common. Most cases (70 to 80%) are post-surgical, while others are a consequence of autoimmunity or are idiopathic. This disease affects approximately 70,000 to 90,000 people in the United States, according to Ascendis.
Palopegteriparatide, developed under the name TransCon PTH, is a prodrug of parathyroid hormone. It is administered as a once-daily subcutaneous injection designed to deliver solid PTH levels within general physiological limits for 24 hours. In contrast, traditional active treatments with vitamin D and calcium are associated with fluctuations in calcium levels, hypercalciuria, renal failure, and reduced quality of life.
The FDA’s approval was based on knowledge of the company’s Phase 2 PaTH Forward trial and Phase 3 PaTHway trial. In both cases, the drug was associated with normalization of serum calcium levels and independence from traditional treatments, with no primary adverse events.
Adverse reactions that occurred in 5% or more of treated patients included injection reactions (39%), vasodilatory symptoms and symptoms (28%), headache (21%), diarrhea (10%), sore throat. back (8%), hypercalcemia (8%) and oropharyngeal pain (7%).
Ascendis is completing manufacturing of an advertising product for the U. S. market and expects to have initial supply in the first quarter of 2025, pending FDA approval of the existing manufactured product, which could come later in 2024.
Miriam E. Tucker is a freelance journalist based in the Washington, DC area. She is a regular contributor to Medscape and has appeared in other work in the Washington Post, NPR’s Shots blog, and Diatribe. She is on X (formerly Twitter). @MiriamETucker.
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