FDA Approves Ascendis Pharma's YORVIPATH for Hypoparathyroidism

Ascendis Pharma has achieved a significant milestone by securing approval from the US Food and Drug Administration (FDA) for its drug YORVIPATH (palopegteriparatide) to treat hypoparathyroidism in adults. Hypoparathyroidism is a rare endocrine disorder characterized by insufficient levels of the parathyroid hormone, impacting various organs in the body.

YORVIPATH is a prodrug of parathyroid hormone (PTH[1-34]) and is designed to be administered once daily. The goal of the treatment is to maintain continuous exposure to released PTH over a 24-hour period, thus addressing the hormone deficiency more effectively. Upon launching YORVIPATH, Ascendis Pharma plans to introduce a comprehensive patient support initiative through its US Ascendis Signature Access Program. This program will provide services to assist patients in managing their treatment process and offer financial aid to those who qualify.

The FDA's approval of YORVIPATH was based on an extensive review of the clinical data submitted with the new drug application for TransCon PTH (palopegteriparatide). This data package included results from global Phase II PaTH Forward and Phase III PaTHway clinical trials. These studies were crucial in demonstrating the drug's efficacy and safety, ultimately leading to its approval.

Ascendis Pharma is currently in the process of finalizing the production of the commercial product for the US market. The company anticipates that the initial supply will be available in the first quarter of 2025. Additionally, Ascendis plans to seek FDA approval to commercialize the already manufactured product. If this request is granted, the drug could be introduced to the US market as early as the fourth quarter of 2024.

Jan Mikkelsen, President and CEO of Ascendis Pharma, expressed his satisfaction with the FDA's approval of YORVIPATH. He emphasized that this approval aligns with the company's commitment to scientific innovation and dedication to addressing the significant unmet medical needs of the hypoparathyroidism community in the US. Mikkelsen also extended his gratitude to the patients, clinicians, and advocates who contributed to reaching this milestone.

In addition to its efforts in the US, Ascendis Pharma has also entered into an exclusive distribution agreement with Pendopharm. This agreement, established in July 2024, aims to make the hypoparathyroidism treatment TransCon PTH available in the Canadian market. This strategic move reflects Ascendis Pharma's broader commitment to expanding access to its innovative treatments for hypoparathyroidism on an international scale.

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