The U.S. Food and Drug Administration (FDA) has approved Ascendis Pharma A/S’s Yorvipath (palopegteriparatide) for the treatment of hypoparathyroidism in adults. This approval marks a significant step forward for individuals living with this rare condition, which affects an estimated 70,000 to 90,000 people in the United States.
Hypoparathyroidism occurs when the parathyroid glands, located in the neck, do not produce enough parathyroid hormone (PTH). This hormone plays a crucial role in regulating calcium levels in the blood. When PTH levels are low, it leads to hypocalcemia (low calcium levels) and hyperphosphatemia (high phosphorus levels), impacting multiple organs and causing a range of symptoms.
Yorvipath, a prodrug of parathyroid hormone (PTH[1-34]), is administered once daily and designed to provide continuous exposure to released PTH over a 24-hour period. This sustained release aims to address the fluctuating calcium levels often experienced by individuals with hypoparathyroidism.
The FDA’s approval was based on the comprehensive clinical data from the global Phase 2 PaTH Forward and Phase 3 PaTHway trials, demonstrating the efficacy and safety of Yorvipath. Ascendis plans to offer a suite of patient services through its Ascendis Signature Access Program, providing support and resources for individuals using Yorvipath.
Ascendis is currently completing the manufacturing of a commercial product for the U.S. market and anticipates an initial supply will be available in the first quarter of 2025. The company is also seeking FDA approval to commercialize an existing manufactured product, which, if approved, could be introduced in the U.S. in the fourth quarter of 2024.
The approval of Yorvipath comes at a crucial time, as Takeda Pharmaceutical Co Ltd is discontinuing the manufacturing of its Natpar/Natpara (parathyroid hormone) for Injection globally by the end of 2024 due to unresolved supply issues. Takeda will not re-commercialize Natpara in the U.S. and will discontinue manufacturing it worldwide.
This move underscores the importance of Ascendis Pharma’s Yorvipath, which now fills a crucial gap in treatment options for patients with hypoparathyroidism. Ascendis Pharma’s shares rose by 4.45% to $132.62 on Monday following the FDA’s approval.
