U.S. FDA Orphan Drug exclusivity provides seven years of market exclusivity for YORVIPATH in the United States for the treatment of hypoparathyroidism in adults

Ascendis Pharma A/S  announced that the United States Food & Drug Administration (FDA) has granted Orphan Drug exclusivity to YORVIPATH^® (palopegteriparatide, developed as TransCon PTH), providing seven years of market exclusivity for YORVIPATH in the United States for the treatment of hypoparathyroidism in adults. YORVIPATH is a prodrug of parathyroid hormone (PTH [1-34]), 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 United States.

“YORVIPATH has now been granted orphan exclusivity in the U.S., European Union, and other countries, reflecting the uniqueness of YORVIPATH to address this rare disease,” said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. “As the first and only FDA-approved treatment of hypoparathyroidism in adults, we believe that YORVIPATH has great potential to address the underlying disease and look forward to making it available to patients in the U.S. as quickly as possible.”

TransCon PTH (palopegteriparatide) originally received Orphan Drug Designation from the U.S. FDA in June 2018. The FDA grants orphan designation to drugs that are intended for the safe and effective treatment, diagnosis, or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States, and that potentially may be safer or more effective than already approved products. 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, waiver of FDA user fees, and tax credits for clinical research. 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.