Ascendis Pharma Granted U.S. Orphan Drug Exclusivity for YORVIPATH® in Adult Hypoparathyroidism

Ascendis Pharma A/S has announced that the U.S. Food and Drug Administration (FDA) has awarded Orphan Drug exclusivity to YORVIPATH® (palopegteriparatide, developed as TransCon PTH). This approval grants YORVIPATH seven years of market exclusivity in the United States for the treatment of hypoparathyroidism in adults. Hypoparathyroidism is a rare endocrine disorder characterized by insufficient levels of parathyroid hormone (PTH), affecting an estimated 70,000 to 90,000 individuals in the U.S.

YORVIPATH is a once-daily administered prodrug of parathyroid hormone, designed to ensure continuous exposure to released PTH over a 24-hour period, thereby addressing the hormone deficiency impacting multiple organs in patients with hypoparathyroidism. The condition, which is primarily caused by post-surgical complications, leads to severe and potentially life-threatening complications, such as neuromuscular irritability, renal issues, extra-skeletal calcifications, and cognitive impairment.

Jan Mikkelsen, President and CEO of Ascendis Pharma, highlighted the significance of this FDA approval. He emphasized that YORVIPATH is the first and only FDA-approved treatment for adult hypoparathyroidism and underscored the company's commitment to making this innovative treatment available to patients in the U.S. as soon as possible. YORVIPATH has also been granted orphan exclusivity in the European Union and other countries, reflecting its unique capability to address this rare disease.

Palopegteriparatide, the active compound in YORVIPATH, initially received Orphan Drug Designation from the U.S. FDA in June 2018. This designation is awarded to drugs aimed at treating, diagnosing, or preventing rare diseases that affect fewer than 200,000 people in the United States. The Orphan Drug Designation provides several benefits to the drug developer, including a seven-year period of marketing exclusivity in the U.S. upon product approval, waiver of FDA user fees, and tax credits for clinical research.

It is important to note that the granting of Orphan Drug Designation does not change the FDA's requirements for establishing the safety and effectiveness of the drug through well-controlled studies to support approval and commercialization. The designation also does not offer any specific advantage during the regulatory review and approval processes.

Ascendis Pharma A/S is leveraging its innovative TransCon technology platform to build a leading biopharma company focused on making meaningful differences in patients' lives. The company is dedicated to using its TransCon technologies to develop new and potentially best-in-class therapies. Based in Copenhagen, Denmark, Ascendis operates additional facilities in Europe and the United States.

Hypoparathyroidism results from insufficient levels of parathyroid hormone, a critical regulator of calcium and phosphate balance in the body. This hormone acts directly on bones and kidneys and indirectly on the intestines. The majority of hypoparathyroidism cases are due to post-surgical complications, although other causes include autoimmune and idiopathic conditions.

Ascendis Pharma’s mission, guided by its core values of Patients, Science, and Passion, is to apply its TransCon technology platform to develop therapies that can significantly improve patients' lives. The company's progress with YORVIPATH marks a significant step forward in addressing the needs of those suffering from hypoparathyroidism.