FDA Approves Ascendis' Yorvipath for Hypoparathyroidism

The FDA has granted approval to Ascendis Pharma’s Yorvipath (palopegteriparatide) for treating hypoparathyroidism in adults, with the decision arriving slightly ahead of a recently revised timeline. Ascendis Pharma highlighted that this injectable, once-daily therapy, formerly known as TransCon PTH, is the first treatment authorized in the United States for this specific condition.

Earlier this year, the decision on Yorvipath faced a delay in May when the FDA announced it required additional time to review new information submitted by Ascendis, which the agency categorized as a major amendment to the existing application. This decision followed a complete response letter issued by the FDA a year prior, which pointed out issues related to the manufacturing control strategy, specifically the variability in the delivered dose of the drug/device combination.

The endorsement by the FDA comes on the heels of a similar approval in Europe last November. The green light in the United States was supported by data from the Phase III PaTHway trial and the mid-stage PaTH Forward trials. In these studies, 79% and 86% of patients, respectively, achieved normalization of serum calcium levels and independence from conventional therapy after six months of treatment.

Ascendis Pharma plans to launch Yorvipath in the US in the first quarter of 2025, as it continues efforts to complete the manufacturing of the commercial product. However, the company also indicated its intention to seek FDA approval to market the already manufactured product, which could potentially expedite the launch to the next quarter.

Yorvipath is a prodrug of the parathyroid hormone (PTH[1-34]), engineered to provide continuous exposure to released PTH over a 24-hour dosing period. This design aims to offer a more stable and effective treatment option for adult patients suffering from hypoparathyroidism, a condition characterized by insufficient production of parathyroid hormone, leading to low levels of calcium in the blood and high levels of phosphorus.

Hypoparathyroidism is a rare endocrine disorder that can significantly affect a patient's quality of life. Current treatment options are limited and typically involve calcium and active vitamin D supplements, which do not adequately replicate the natural PTH regulation. Yorvipath’s approval represents a significant advancement in addressing this unmet medical need by potentially offering a more precise and consistent management of the condition.

In summary, the FDA's approval of Yorvipath for adults with hypoparathyroidism marks a pivotal step forward in the treatment landscape of this rare disorder. The approval, supported by robust clinical trial data, underscores the drug's potential to normalize serum calcium levels and reduce dependence on conventional therapies. As Ascendis Pharma prepares for its commercial launch, patients and healthcare providers alike anticipate the availability of this innovative therapy.