YORVIPATH® Becomes The First and Only FDA-Approved Treatment For Hypoparathyroidism In Adults

Ascendis Pharma has announced that the U.S. Food and Drug Administration (FDA) has granted approval for YORVIPATH® (palopegteriparatide, previously known as TransCon PTH) to treat adults with hypoparathyroidism. This innovative treatment acts as a prodrug for parathyroid hormone (PTH[1-34]) and is designed for once-daily administration, ensuring a steady release of PTH throughout the 24-hour period. Hypoparathyroidism is a rare endocrine disorder characterized by inadequate levels of parathyroid hormone, affecting various organs and impacting approximately 70,000 to 90,000 individuals in the United States.

Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer, said in a statement, “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. We are deeply grateful to patients, clinicians, and advocates for their many contributions to this important milestone.”

Upon the launch of YORVIPATH, Ascendis intends to provide a range of patient services through its U.S. Ascendis Signature Access Program (A.S.A.P.). This program will include guidance throughout the treatment process and financial assistance options for qualifying patients.

Patty Keating, Executive Director of the HypoPARAthyroidism Association, added “FDA approval of YORVIPATH is such an important milestone for our community. 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 consequences of hypoparathyroidism on the health and quality of life of our patients can be extraordinarily debilitating. The ability to address the underlying cause of this disease is crucial and will be an important advancement for our patients with hypoparathyroidism,” 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 FDA's approval of YORVIPATH was founded on its evaluation of the clinical data provided in the Company's New Drug Application for TransCon PTH (palopegteriparatide). This included findings from the international Phase 2 PaTH Forward trial and the Phase 3 PaTHway study. Ascendis is finalizing the production of its commercial product for the U.S. market and expects the first supply to be ready by the first quarter of 2025. Furthermore, the company intends to seek FDA approval to market its already manufactured product, which, if granted, could be launched in the U.S. by the fourth quarter of 2024.