Ascendis Pharma should be well prepared for the U.S. launch of its hormone replacement therapy for hypoparathyroidism, which was approved on Friday by the FDA.
After all, the company was initially set to bring the drug to the market 15 months ago before the FDA rejected it with a surprise complete response letter (CRL). Then three months ago, Ascendis again was ready to roll before the U.S. regulator said it needed an additional three months to make its decision.
Finally, Copenhagen-based Ascendis is now good to go with once-daily Yorvipath (palopegteriparatide), also known as TransCon PTH, which is the first FDA-approved product for hypoparathyroidism in adults.
“We have worked on this product for nearly 10 years,” Jan Mikkelsen, the CEO of Ascendis, said during a conference call Monday. “We’re looking forward to being in a position that we as fast as possible can move this product opportunity out to patients.”
Still, Yorvipath won’t be immediately available. Ascendis expects the initial supply to be available in the first quarter of next year with the potential to move the launch up to the fourth quarter this year, as the company has requested FDA approval to commercialize doses it has already made.
But manufacturing was the issue 15 months ago when the agency cited concerns in the CRL about Ascendis’ production control strategy because of variability found in delivered doses.
“We’ve had infrastructure in place in the U.S., ready to launch a second product for some time,” Scott Smith, chief financial officer of Ascendis, said.
Meanwhile, the European Union signed off on Yorvipath in November 2023, and the U.K. followed suit three months ago. In Germany and Austria, where Yorvipath was first launched, there are more than 250 patients taking the treatment, the company said.
On Monday, Mikkelsen said Ascendis “has not decided” how to price Yorvipath in the U.S., asking investors to stay tuned for its next quarterly presentation for “more clarity.”
In a note to clients, ISI Evercore modeled a $150,000 to $160,000 annual price for Yorvipath, but analysts "wouldn't be surprised to see Ascendis' price higher." They also project 2025 sales of Yorvipath to reach $100 million in the US and $200 million worldwide, which assumes 1,300 to 1,500 US patients on the treatment by the end of the year.
Hypoparathyroidism, which affects between 70,000 and 90,000 people in the U.S., is an endocrine system disorder in which the parathyroid glands don’t produce enough calcium, causing muscle aches and spasms. Four parathyroid glands in the neck control the critical balance between calcium and phosphorus. The disease can eventually lead to kidney complications and cognitive impairment.
Yorvipath, which provides continuous exposure to released parathyroid hormone (PTH) over a 24-hour dosing period, proved itself in a phase 3 trial. The study showed that the hormone replacement therapy raised the calcium levels in 79% of patients and allowed 95% of patients to be freed from conventional therapy, which includes oral calcium and active vitamin D.
Yorvipath marks Ascendis’ second product. Skytrofa, a long-acting growth hormone for children, was endorsed in 2021. Last year, sales for the once-weekly medicine reached 179 million euros ($194 million). The company projects Skytrofa sales will reach 320 million euros ($349 million) to 340 million euros ($371 million) this year.