FDA Approves Ascendis Pharma’s Yorvipath as the First Treatment for Adults with Hypoparathyroidism

Image Credit: Adobe Stock Images/Maxim Borbut

The FDA has approved Ascendis Pharma's Yorvipath (palopegteriparatide) as the first and only treatment for hypoparathyroidism. According to the company, approval was based on positive results from the global Phase II PaTH Forward and Phase III PaTHway trials. Yorvipath is classified as a once-daily prodrug of parathyroid hormone (PTH [1-34]) designed to provide continuous PTH exposure over 24 hours, addressing a significant unmet need in treating patients with hypoparathyroidism.¹

“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, president, CEO, Ascendis Pharma, in a press release. “We are deeply grateful to patients, clinicians, and advocates for their many contributions to this important milestone.”

Moving forward, Ascendis plans to support patients through its United States Ascendis Signature Access Program, aiming to have commercial supply ready by Q1 2025. Additionally, the company stated that it may also seek approval to introduce existing manufactured products in Q4 2024, aiming to accelerate patient access.¹

“FDA approval of Yorvipath is such an important milestone for our community,” said Patty Keating, executive director, HypoPARAthyroidism Association, in the press release. “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.”

Ascendis warns against the use of Yorvipath in patients who are at increased risk of osteosarcoma, such as patients with open epiphyses, metabolic bone diseases other than hypoparathyroidism, unexplained elevations of alkaline phosphatase, bone metastases, history of skeletal malignancies, history of external beam or implant radiation therapy involving the skeleton, and hereditary disorders predisposing to osteosarcoma. Serious adverse events (AEs) such as hypercalcemia requiring hospitalization have been reported when patients have started or increased dosage of Yorvinpath.¹

In the United States, hypoparathyroidism effects anywhere from 70,000 to 90,000 people. Ascendis reports that 70% to 80% of hypoparathyroidism results from post-surgical complications, with autoimmune and idiopathic causes also accounting for a number of cases.¹

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

Reference

FDA Approves YORVIPATH® (Palopegteriparatide) as the First and Only Treatment for Hypoparathyroidism in Adults. Ascendis Pharma. August 12, 2024. Accessed August 12, 2024. https://investors.ascendispharma.com/news-releases/news-release-details/fda-approves-yorvipathr-palopegteriparatide-first-and-only