This week, the U.S. Food and Drug Administration (FDA) granted market approval to Ascendis Pharma A/S (NASDAQ: ASND), a Denmark-based pharmaceutical company specializing in rare diseases, for its hypoparathyroidism treatment, palopegteriparatide. The therapy, to be marketed as Yorvipath, is a once-daily prodrug of parathyroid hormone (PTH[1-34]) designed to address the deficiency of PTH in patients with hypoparathyroidism over a 24-hour period.
The approval follows a 15-month journey after Ascendis initially received a complete response letter (CRL) from the FDA due to manufacturing concerns. With this green light, Yorvipath becomes accessible to an estimated 70,000 to 90,000 individuals in the U.S. living with hypoparathyroidism. Ascendis’s product steps in to fill a significant void left by the withdrawal of Takeda’s Natpar/Natpara at the start of this year, which had been affected by manufacturing challenges, including the formation of rubber particles, impacting its 2,800 regular users in the U.S.
In China, Visen Pharmaceuticals Co., Ltd, a joint venture established in 2018 between Ascendis and Chinese investors, is developing Yorvipath. This week, Visen also reported positive results from the Phase III PaTHway China Trial, where palopegteriparatide met all primary and secondary endpoints in treating adults with hypoparathyroidism. A remarkable 77.6% of patients in the trial were able to manage their condition independently of standard treatments, requiring minimal vitamin D and calcium supplements. Visen is poised to submit a New Drug Application (NDA) in China at the earliest opportunity.- Flcube.com
