Ascendis Receives a Grudging FDA Approval for Yorvipath, a Hormone Replacement Treatment

Ascendis Pharma should be in a good position to launch its hormone replacement therapy for hypoparathyroidism in the U.S. market, as it received FDA approval on Friday.

The company had initially expected to launch the drug 15 months ago, but the FDA issued a Complete Response Letter (CRL), which came as a complete shock to the firm. Three months ago, Ascendis was once again ready to approach the U.S. regulator, only to be told that they needed another three months to reach a final decision.

However, Yorvipath’s educational programs will not be launched immediately. The company believes that the initial supply of the drug will be ready in Q1 of the following year. They have requested FDA approval to launch the actual doses as early as Q4 of this year.

The primary concern, 15 months ago, was related to manufacturing. The FDA had raised issues with production control in its CRL due to variability in the delivered doses.

“We have had the supporting infrastructure in the U.S. in place for quite some time in anticipation of the second product,” said Scott Smith, the Chief Financial Officer of Ascendis.

Meanwhile, Yorvipath was approved by the European Union in November 2023, and the United Kingdom followed suit three months ago. The company revealed that over 250 patients are currently using the drug in Germany and Austria, where Yorvipath was first introduced.

According to a client note by ISI Evercore, Yorvipath is expected to be priced between $150,000 and $160,000 annually. Analysts also believe that Ascendis may set a higher price. Sales of Yorvipath are projected to reach $100 million in the U.S. and $200 million globally by 2025, assuming there are 1,300-1,500 patients treated.

Hypoparathyroidism, which affects between 70,000 and 90,000 people in the U.S., is an endocrine disorder where the parathyroid glands fail to produce enough calcium. This results in muscle rigidity and spasms. The four glands in the neck regulate the calcium-to-phosphorus ratio, which is essential for life. The disease is known to cause kidney problems and also affects cognitive function.

In a Phase 3 trial, Yorvipath, which delivers parathyroid hormone (PTH) to patients at least once every 24 hours, showed promising results. The research indicated an average 79% increase in serum calcium levels during hormone replacement therapy. Furthermore, 95% of patients were able to discontinue traditional treatments, including oral calcium and active vitamin D.

Yorvipath is Ascendis’ second product. Its first, SKYTROKA, a once-weekly, long-acting recombinant human growth hormone for children, was approved in 2021. Last year, SKYTROKA generated €179 million ($194 million) in sales, and the company projects that sales will increase to between €320 million ($349 million) and €340 million ($371 million) this year.

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