Ascendis Pharma's hormone disorder therapy gets US approval

By Sneha S K and Sriparna Roy

(Reuters) -Ascendis Pharma said on Monday the U.S. Food & Drug Administration had approved its therapy to treat adult patients with a hormone disorder, sending the Danish drugmaker's shares more than 10% higher in premarket trading.

Ascendis's Yorvipath, will become the only approved treatment for the condition, called hypoparathyroidism, in the United States after rival Takeda stops manufacturing its injection, Natpara, at the end of this year due to supply issues.

The approval for Yorvipath comes after a couple of setbacks - including an extension of FDA review in May, and a rejection of approval last year, on concerns linked to manufacturing controls of the drug and device combination.

The company's mid- and late-stage studies showed the majority of the patients treated with Yorvipath did not need to take vitamin D and calcium supplements daily - which are the current standard-of-care treatments for hypoparathyroidism.

It is a rare endocrine disease caused by low levels of parathyroid hormone that impact multiple organs and affects an estimated 70,000 to 90,000 people in the United States, according to the company.

Low production of parathyroid hormone in hypoparathyroidism leads to abnormally low calcium levels in the blood and an increase of phosphorus.

Ascendis anticipates initial supply of the once-daily therapy will be available in the first quarter of 2025, and expects to announce the annual price of the therapy in the coming week.

Evercore ISI analyst Gavin Clark-Gartner estimates peak sales to be about $2.5 billion on an adjusted basis and expects the therapy to have a market penetration of about 12.5% in the United States.

Ascendis also plans to request FDA's approval to commercialize existing manufactured products qualified for other markets, which if approved, could be introduced in the United States in the fourth quarter of 2024.

(Reporting by Sriparna Roy and Sneha S K in Bengaluru; Editing by Shinjini Ganguli)