Ascendis Pharma bags YORVIPATH FDA approval for adult hypoparathyroidism

In a landmark decision, the U.S. Food and Drug Administration (FDA) has granted approval to Ascendis Pharma A/S’s YORVIPATH (palopegteriparatide), making it the first and only treatment specifically for adult hypoparathyroidism. This rare endocrine disorder, characterised by insufficient levels of parathyroid hormone, affects between 70,000 to 90,000 people across the United States. YORVIPATH, developed under the name TransCon PTH, represents a significant advancement in addressing this challenging condition.

Breakthrough Treatment for Hypoparathyroidism

YORVIPATH is a prodrug designed to provide continuous exposure to parathyroid hormone (PTH[1-34]) over a 24-hour period with a once-daily administration. This innovative approach aims to mitigate the debilitating effects of hypoparathyroidism by ensuring steady hormone levels, which is crucial for managing the disease’s impact on multiple organs.

Ascendis Pharma’s President and Chief Executive Officer, Jan Mikkelsen, expressed enthusiasm about the approval, highlighting the company’s dedication to advancing treatment for this underserved patient population. Mikkelsen underscored the commitment to addressing significant medical needs and expressed gratitude to the patients, clinicians, and advocates who contributed to this milestone.

Patient Support Services

At the launch of YORVIPATH, Ascendis Pharma will introduce the U.S. Ascendis Signature Access Program (A.S.A.P.), which will offer extensive patient support services. These include assistance with navigating the treatment journey and financial aid programs for eligible individuals, aiming to ease access to this new therapy.

Patty Keating, Executive Director of the HypoPARAthyroidism Association, celebrated the approval as a crucial development for the hypoparathyroidism community. She acknowledged the importance of recognising the seriousness of the condition and anticipated that YORVIPATH would provide a valuable alternative to conventional therapies.

Clinical Trials and Future Availability

The FDA’s decision was based on a thorough review of clinical data from Ascendis Pharma’s New Drug Application, which included results from the global Phase 2 PaTH Forward and Phase 3 PaTHway trials. Dr. Lynn Kohlmeier, an endocrinologist and investigator in the PaTHway Trial, highlighted the importance of addressing the underlying cause of hypoparathyroidism and viewed YORVIPATH as a crucial advancement for patient care.

Ascendis Pharma is currently finalising the manufacturing process for the U.S. market. The company anticipates that initial supplies of YORVIPATH will be available in the first quarter of 2025. Additionally, Ascendis plans to seek FDA approval for existing manufactured products, potentially introducing them to the U.S. market in the fourth quarter of 2024.