Ascendis Pharma's YORVIPATH Earns U.S. Orphan Drug Status

Ascendis Pharma A/S has secured Orphan Drug exclusivity from the U.S. Food & Drug Administration (FDA) for its therapy, YORVIPATH® (palopegteriparatide, developed under the name TransCon PTH). This approval grants the company seven years of exclusive rights to market YORVIPATH in the U.S. as a treatment for adult hypoparathyroidism, a rare endocrine disorder.

YORVIPATH, a prodrug of parathyroid hormone (PTH [1-34]), is administered once daily and is designed to maintain consistent PTH levels throughout the day. Hypoparathyroidism affects an estimated 70,000 to 90,000 individuals in the U.S. and disrupts various bodily functions due to inadequate parathyroid hormone production

Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer stated, “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. 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.”

In June 2018, the U.S. FDA awarded Orphan Drug Designation to TransCon PTH (palopegteriparatide). This designation is reserved for medications aimed at treating, diagnosing, or preventing rare diseases affecting fewer than 200,000 individuals in the U.S., and which may offer improved safety or effectiveness compared to existing treatments.

While Orphan Drug Designation provides several advantages, such as a seven-year period of market exclusivity in the U.S. once the drug is approved, exemption from FDA user fees, and tax credits for clinical research, it does not change the FDA's requirements for demonstrating a drug's safety and efficacy through rigorous studies. Additionally, it does not confer any special benefits during the regulatory review and approval process.