FDA Approves Ascendis’ Skytrofa for Adults with Growth Hormone Deficiency

Rare Daily Staff

The U.S. Food and Drug Administration has approved Ascendis Pharma’s Skytrofa for the replacement of endogenous growth hormone in adults with growth hormone deficiency, a rare disorder resulting from decreased or total loss of growth hormone production.

Growth hormone deficiency (GHD) is characterized by short stature and metabolic complications. Though crucial for growth in children, growth hormone is also important in adults for metabolism, bone and muscle maintenance, and heart health.

Skytrofa, first approved for children one year of age and older with growth failure due to inadequate secretion of endogenous growth hormone, is a long-acting prodrug of somatropin. It was the first FDA-approved treatment utilizing Ascendis Pharma’s TransCon technology.

TransCon molecules have three components: an unmodified parent drug, an inert carrier that protects it, and a linker that temporarily binds the two. When bound, the carrier inactivates and shields the parent drug from clearance. Upon injection into the body, physiological conditions initiate the release of the active, unmodified parent drug in a predictable manner. Because the parent drug remains unmodified, its original mode of action is expected to be maintained.

The FDA’s approval of Skytrofa for adult GHD was based on results from foresiGHt, a phase 3, randomized, parallel-arm, placebo-controlled, and open-label clinical trial that compared the efficacy and safety of weekly TransCon hGH with those of weekly placebo and daily somatropin in adults with GHD.

“The abnormal body composition, dyslipidemia, and insulin resistance that are the hallmarks of adult GHD predispose affected individuals to serious medical complications such as metabolic syndrome, increased cardiovascular risk, and impaired quality of life—including cognitive dysfunction, depression, anxiety, sleep disturbances, and reduced physical and mental drive,” said Kevin Yuen of the Barrow Neurological Institute, University of Arizona College of Medicine, and Creighton University School of Medicine in Phoenix, Arizona. “Despite this, adherence to daily somatropin injections, which have been the standard of care for more than 25 years, remains a problem. It is no surprise that patients are looking for a less burdensome treatment regimen and a new treatment option such as Skytrofa.”