FDA approves Ascendis drug for hypoparathyroidism

The FDA has approved Ascendis Pharma’s palopegteriparatide, marketed under the brand name Yorvipath, as the first treatment for hypoparathyroidism in adults. Yorvipath is a prodrug of parathyroid hormone (PTH[1-34]) that is designed to deliver continuous exposure to PTH over a 24-hour period through once-daily administration. Hypoparathyroidism is a rare endocrine disorder characterized by insufficient levels of parathyroid hormone, which affects multiple organs and impacts an estimated 70,000 to 90,000 individuals in the United States.

The approval of Yorvipath was based on clinical data derived from the phase 2 PaTH Forward and phase 3 PaTHway trials, which were part of Ascendis Pharma’s New Drug Application (NDA). Initially, the drug was rejected by the FDA in 2023 due to concerns about the manufacturing control strategy for variability in the delivered dose of the drug/device combination product. After discussions with the FDA, Ascendis resubmitted its NDA in November. Although the agency extended its review period last May, Yorvipath has now been approved, making it the only treatment available that specifically targets the underlying cause of hypoparathyroidism.

Ascendis Pharma is gearing up for the commercial release of Yorvipath in the U.S., with the first supply anticipated in the first quarter of 2025. As part of the product launch, Ascendis plans to offer patient services through its U.S. Ascendis Signature Access Program (A.S.A.P.). This program will include support for navigating treatment and financial assistance for eligible patients.