"Ascendis Pharma's hormone therapy for disorder wins US approval"

Ascendis Pharma's hormone therapy for a rare disorder has received approval from the US FDA, representing a major advancement in treatment options for patients with hormone-related conditions. This approval highlights the therapy's potential to improve patient outcomes and marks a significant achievement for the company in its mission to address unmet medical needs.

‍August 12 (Reuters) - Ascendis Pharma announced on Monday that the U.S. Food & Drug Administration has approved its therapy for treating adult patients with a hormone disorder, causing the Danish company's shares to rise over 10% in premarket trading. Yorvipath, Ascendis's new treatment, will be the only FDA-approved option for hypoparathyroidism in the U.S. after Takeda (4502.T) stops producing its injection, Natpara, at the end of the year due to supply issues.

This approval follows a series of challenges, including an FDA review extension in May and a previous rejection last year, mainly due to concerns about manufacturing controls for the drug-device combination. Studies conducted by Ascendis in mid- and late-stage trials demonstrated that most patients treated with Yorvipath did not require daily vitamin D and calcium supplements, which are the current standard treatment for hypoparathyroidism.

Hypoparathyroidism is a rare endocrine disorder affecting 70,000 to 90,000 people in the U.S., caused by insufficient production of parathyroid hormone, leading to low blood calcium levels and high phosphorus levels. Ascendis expects the initial supply of the once-daily therapy to be available by the first quarter of 2025 and plans to announce the therapy's annual price within the next week.

Evercore ISI analyst Gavin Clark-Gartner predicts peak sales of about $2.5 billion, with the therapy potentially capturing a 12.5% market share in the U.S. Ascendis also intends to seek FDA approval to market existing products manufactured for other regions, potentially launching them in the U.S. in the fourth quarter of 2024.

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Source: Reuters‍