Ascendis Pharma’s yorvipath granted orphan drug exclusivity in U.S.

Ascendis Pharma announced that the FDA has granted Orphan Drug exclusivity to Yorvipath, providing seven years of market exclusivity for Yorvipath in the U.S. for the treatment of hypoparathyroidism in adults. Yorvipath is a prodrug of parathyroid hormone administered once daily, designed to provide continuous exposure to released PTH over the 24-hour dosing period. Hypoparathyroidism is a rare endocrine disease caused by insufficient levels of parathyroid hormone that impact multiple organs and affects an estimated 70,000 to 90,000 people in the U.S. Subject to certain exceptions, orphan designation provides a drug developer with certain benefits and incentives, including a seven-year period of U.S. marketing exclusivity upon approval of the product in the orphan-designated indication. The granting of orphan designation does not alter the FDA’s regulatory requirements to establish safety and effectiveness of a drug through adequate and well-controlled studies to support approval and commercialization, nor does it provide any advantage during the regulatory review and approval processes.