Hypoparathyroidism is a rare endocrine disease with multi-organ impacts affecting an estimated 70,000 to 90,000 people in the United States

Ascendis Pharma A/S announced that the U.S. Food & Drug Administration (FDA) has approved YORVIPATH^® (palopegteriparatide; developed as TransCon PTH) for the treatment of hypoparathyroidism in adults. YORVIPATH is a prodrug of parathyroid hormone (PTH[1-34]), administered once daily, designed to provide continuous exposure to released PTH over the 24-hour dosing period. Hypoparathyroidism is a rare endocrine disease caused by insufficient levels of parathyroid hormone that impact multiple organs and affects an estimated 70,000 to 90,000 people in the United States.

“FDA approval of our second TransCon product, YORVIPATH, reflects our values and dedication to following the science to help patients, as well as our unwavering commitment these past years to addressing the significant unmet medical needs of the hypoparathyroidism community in the United States,” said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. “We are deeply grateful to patients, clinicians, and advocates for their many contributions to this important milestone.”

At launch, Ascendis plans to offer a suite of patient services for YORVIPATH through its U.S. Ascendis Signature Access Program (A.S.A.P.), including support navigating the treatment journey and financial assistance programs for eligible patients.

“FDA approval of YORVIPATH is such an important milestone for our community,” said Patty Keating, Executive Director of the HypoPARAthyroidism Association. “We are thankful that the seriousness of our condition has been understood and our voices heard. We look forward to having this new treatment option to help us move beyond the limits and risks of conventional therapy.”

The FDA based its approval of YORVIPATH on their review of the clinical package for TransCon PTH (palopegteriparatide) submitted with the Company’s New Drug Application, including data from the global Phase 2 PaTH Forward and Phase 3 PaTHway trials.

“The consequences of hypoparathyroidism on the health and quality of life of our patients can be extraordinarily debilitating,” said Lynn Kohlmeier, M.D., endocrinologist at Spokane Osteoporosis & Endocrinology, Chair of the Medical Advisory Board of the HypoPARAthyroidism Association, and an investigator in the PaTHway Trial. “The ability to address the underlying cause of this disease is crucial and will be an important advancement for our patients with hypoparathyroidism.”

Ascendis is completing manufacturing of commercial product for the U.S. market and anticipates initial supply will be available in the first quarter of 2025. In addition, Ascendis plans to request FDA approval to commercialize existing manufactured product, which, if approved, could be introduced in the U.S. in the fourth quarter of 2024.