FDA approves Ascendis Pharma’s Skytrofa for adult growth hormone deficiency

Published 28/07/2025, 12:06
FDA approves Ascendis Pharma’s Skytrofa for adult growth hormone deficiency

COPENHAGEN - The U.S. Food and Drug Administration has approved Ascendis Pharma A/S’s (NASDAQ:ASND) Skytrofa for the treatment of growth hormone deficiency in adults, the company announced Monday. The biotech company, currently valued at $10.14 billion, has seen its stock surge nearly 30% over the past six months, according to InvestingPro data.

Skytrofa (lonapegsomatropin-tcgd) is a once-weekly injection that provides sustained release of active, unmodified somatropin. The drug was previously approved in 2021 for pediatric growth hormone deficiency. With an impressive gross profit margin of 85.3% and revenue growth of 12.06% in the last twelve months, Ascendis continues to demonstrate strong commercial execution.

The FDA’s approval was based on results from the Phase 3 foresiGHt clinical trial, which compared the efficacy and safety of weekly Skytrofa with weekly placebo and daily somatropin in adults with growth hormone deficiency.

Growth hormone deficiency in adults can lead to abnormal body composition, dyslipidemia, insulin resistance, and increased cardiovascular risk, as well as impaired quality of life including cognitive dysfunction and depression.

"It is no surprise that patients are looking for a less burdensome treatment regimen, and a new treatment option such as Skytrofa, with once-weekly injection and its unique release of unmodified somatropin, is expected to help improve both real-world adherence and overall outcomes," said Kevin Yuen, M.D., of Barrow Neurological Institute. InvestingPro analysts share this optimism, with six analysts recently revising their earnings estimates upward, and the consensus price target suggesting significant upside potential.

Jan Mikkelsen, President and CEO of Ascendis Pharma, called the approval "the first of many planned label expansions" as the company aims to become a leader in treating rare endocrine disorders.

The company stated it plans to initiate basket trials for idiopathic short stature, SHOX deficiency, Turner syndrome, and small for gestational age, as well as combination therapy trials in achondroplasia and hypochondroplasia, in the fourth quarter of 2025. For investors seeking deeper insights into Ascendis Pharma’s growth potential and financial health, InvestingPro offers comprehensive analysis and additional ProTips in its detailed research report, part of its coverage of over 1,400 US equities.

Common side effects of Skytrofa in adults include swelling due to fluid build-up and low thyroid hormone levels, according to the press release statement.

In other recent news, Ascendis Pharma has reported positive three-year data from its Phase 3 PaTHway Trial for TransCon PTH, showing sustained benefits in adults with hypoparathyroidism. The data, presented at the ENDO 2025 conference, highlighted improvements in biochemical markers, kidney function, and quality of life. Morgan Stanley has assumed coverage of Ascendis Pharma with an Overweight rating, citing a promising commercial trajectory for Yorvipath, a treatment for hypoparathyroidism. UBS reiterated its buy rating, emphasizing the strong launch of Yorvipath and significant demand among moderate to severe patient populations. Oppenheimer raised its price target for Ascendis Pharma to $224, noting strong early demand for Yorvipath based on a survey of U.S. endocrinologists. TD Cowen maintained a Buy rating, following Ascendis Pharma’s positive 26-week data from their Phase 2 COACH trial involving children with achondroplasia. The trial showed improvements in growth velocity and body proportionality, suggesting potential for the combination therapy of TransCon CNP and Skytrofa. These developments reflect ongoing interest and confidence in Ascendis Pharma’s treatment offerings.

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