Ascendis secures FDA approval for hormone therapy Yorvipath

The FDA has granted approval for Ascendis Pharma's hormone replacement therapy, Yorvipath, aimed at treating hypoparathyroidism. This approval follows a 15-month hiatus after an initial rejection due to concerns over the manufacturing process. Yorvipath, a once-daily therapy also known as TransCon PTH (palopegteriparatide), is now the first FDA-approved treatment for adult hypoparathyroidism.

Ascendis Pharma had been poised to launch Yorvipath 15 months prior, only to be halted by the FDA’s complete response letter (CRL), which highlighted issues in the company's production control strategy due to variability in drug doses. Despite these setbacks, Ascendis has remained focused on bringing Yorvipath to market. Three months ago, another potential launch was delayed as the FDA requested additional time to finalize their decision. Now, with approval in hand, Ascendis is gearing up for the U.S. market introduction of Yorvipath.

CEO Jan Mikkelsen expressed enthusiasm during a recent conference call, citing nearly a decade of development efforts. He emphasized the company's readiness to distribute the drug to patients as quickly as possible. However, initial availability won't be immediate. Ascendis anticipates that the first batch of Yorvipath will be ready by the first quarter of the following year, although they are seeking FDA approval to commercialize doses they have already produced, which could potentially move the launch up to the fourth quarter of this year.

Scott Smith, Ascendis’ Chief Financial Officer, mentioned that the company already had the necessary infrastructure in place in the U.S. for launching a second product. This infrastructure readiness underscores the company's preparedness for Yorvipath's introduction.

In Europe, Yorvipath received approval from the European Union in November 2023, followed by the U.K. three months later. The therapy was initially launched in Germany and Austria, where it has already been administered to over 250 patients. Despite the approvals, the company has yet to finalize the pricing for Yorvipath in the U.S., with more details expected in their next quarterly update. Analysts at ISI Evercore have speculated an annual price range of $150,000 to $160,000 for Yorvipath, though they wouldn't be surprised if the final price is higher. Projections indicate that U.S. sales of Yorvipath could reach $100 million by 2025, with global sales potentially hitting $200 million, assuming roughly 1,300 to 1,500 U.S. patients are on the therapy by the end of the year.

Hypoparathyroidism, affecting between 70,000 and 90,000 people in the U.S., is a disorder where the parathyroid glands fail to produce sufficient calcium, leading to muscle aches and spasms. This condition can result in serious complications such as kidney issues and cognitive impairment. The parathyroid glands are crucial for maintaining the balance between calcium and phosphorus in the body.

Yorvipath addresses this imbalance by providing continuous exposure to parathyroid hormone (PTH) over a 24-hour period. In a phase 3 trial, the therapy successfully raised calcium levels in 79% of patients, with 95% of participants able to discontinue conventional treatments, which typically include oral calcium and active vitamin D supplements.

Yorvipath is Ascendis’ second major product release. The company previously launched Skytrofa, a long-acting growth hormone therapy for children, in 2021. Skytrofa saw impressive sales last year, reaching 179 million euros ($194 million). Ascendis projects that Skytrofa’s sales will continue to grow, potentially reaching between 320 million euros ($349 million) and 340 million euros ($371 million) this year.