Ascendis Pharma has received approval from the US Food and Drug Administration for its proprietary prodrug Yorvipath.
The therapeutic is intended to be administered once daily as a monotherapy for hypoparathyroidism in adults.
The FDA’s decision stems from the Phase II PaTH Forward and Phase III PaTHway trials, which demonstrated the efficacy of the drug in the patient population.
Hypoparathyroidism, a rare endocrine disease categorised by dysfunctional levels of the parathyroid hormone, affects approximately 70,000–90,000 people in the US.
When this therapeutic launches in the US, Ascendis plans to offer a range of patient services through its Signature Access Programme, which will include support during the treatment journey, as well as financial assistance initiatives for patients.
“The consequences of hypoparathyroidism on the health and quality of life of our patients can be extraordinarily debilitating,” said Lynn Kohlmeier, endocrinologist at Spokane Osteoporosis & Endocrinology, Chair of the Medical Advisory Board of the HypoPARAthyroidism Association, and an investigator in the PaTHway Trial. “The ability to address the underlying cause of this disease is crucial and will be an important advancement for our patients with hypoparathyroidism.”
“FDA approval of YORVIPATH is such an important milestone for our community,” said the Executive Director of the HypoPARAthyroidism Association. “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 company estimates that the drug will be available on a commercial scale in the first quarter of 2025.
