Ascendis Pharma's Yorvipath Receives Orphan Drug Exclusivity in the U.S. for Hypoparathyroidism

• Ascendis Pharma's Yorvipath (palopegteriparatide) has been granted Orphan Drug Exclusivity by the U.S. FDA for treating hypoparathyroidism in adults.
• The exclusivity provides seven years of market protection in the U.S., preventing similar drugs from being marketed for the same indication.
• Yorvipath is a prodrug of parathyroid hormone (PTH [1-34]) designed for once-daily administration, offering continuous PTH exposure over 24 hours.
• Hypoparathyroidism, affecting 70,000-90,000 people in the U.S., is a rare endocrine disorder caused by insufficient parathyroid hormone levels.

Ascendis Pharma A/S (Nasdaq: ASND) announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug exclusivity to YORVIPATH® (palopegteriparatide), also known as TransCon PTH, for the treatment of hypoparathyroidism in adults. This designation provides seven years of market exclusivity in the United States.

Yorvipath is a prodrug of parathyroid hormone (PTH [1-34]), administered once daily, designed to provide continuous exposure to released PTH over a 24-hour period. The drug aims to address the underlying deficiency in PTH that characterizes hypoparathyroidism.

Impact of Orphan Drug Exclusivity

The FDA grants orphan designation to drugs intended for the safe and effective treatment, diagnosis, or prevention of rare diseases or disorders affecting fewer than 200,000 people in the United States. This designation offers several benefits, including a seven-year period of U.S. marketing exclusivity upon product approval, waiver of FDA user fees, and tax credits for clinical research.

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.”

Understanding Hypoparathyroidism

Hypoparathyroidism is an endocrine disease resulting from insufficient levels of parathyroid hormone (PTH), which is crucial for regulating calcium and phosphate balance. The condition affects an estimated 70,000 to 90,000 people in the United States. Individuals with hypoparathyroidism may experience a range of severe and potentially life-threatening short-term and long-term complications, including neuromuscular irritability, renal complications, extra-skeletal calcifications, and cognitive impairment. Post-surgical hypoparathyroidism accounts for the majority of cases (70-80%), while other etiologies include autoimmune and idiopathic causes.

Ascendis Pharma's TransCon Technology

Ascendis Pharma is leveraging its TransCon technology platform to develop new therapies. The company focuses on creating potentially best-in-class treatments. Ascendis is headquartered in Copenhagen, Denmark, with additional facilities in Europe and the United States.