Ascendis Pharma announced Monday that it won FDA approval for its hypoparathyroidism drug, offering the small community of those suffering from insufficient levels of parathyroid hormone another option, about two years after Takeda pulled its treatment.
Similar to Takeda’s Natpara, which faced manufacturing woes that led to its exit from the market, Ascendis said Yorvipath is a prodrug of parathyroid hormone administered once daily via injection.
Following a rejection in May 2023 and a three-month extension on the FDA’s review in May, Ascendis said the FDA based its approval on the clinical package for TransCon PTH, also known as palopegteriparatide, which includes data from global Phase 2 and Phase 3 trials.
US regulators had delayed their decision on the drug because of what the company said was a “major amendment” to its application. In May, the FDA rejected Ascendis’ application, citing “concerns related to the manufacturing control strategy for variability of delivered dose.”
Ascendis’ win on Monday follows years of back and forth between Takeda and the FDA over their manufacturing issues, which have been ongoing since 2019. In 2018, the last full year of Natpara sales, the treatment brought in about $230 million for Takeda. Since Natpara was recalled in September 2019, no US revenue was recorded.
According to Leerink Partners analysts, Yorvipath is currently approved in Germany and Austria and generated €1.5 million ($1.6 million) in the first quarter of this year. “Yorvipath has a list price of €105K per patient per year in Europe with a discount of ~6%, making Yorvipath ~€98.7K per patient per year,” Leerink added in a note published Monday.
The difficulties with hypoparathyroidism when untreated occur due to low levels of calcium in the blood, according to the National Organization for Rare Disorders. Symptoms can range from the more mild tingling or numbness in the fingers and toes to severe muscle cramps and spasms, as well as breathing issues that can lead to hospitalization, NORD said.
Ascendis’ stock price rose 10% premarket on Monday.
“We are completing manufacturing of commercial product for the U.S. market and anticipate initial supply will be available in the first quarter of 2025,” a company spokesperson said. “In addition, Ascendis plans to request FDA approval to commercialize existing manufactured product qualified for other markets, which, if approved, could be introduced in the U.S. in the fourth quarter of 2024. Annual price will be announced in the coming weeks, with the amount each patient pays dependent on individual insurance plans.”