FDA Approves Ascendis Pharma’s SKYTROFA For Adult Growth Hormone Deficiency

Ascendis Pharma A/S has received U.S. FDA approval for SKYTROFA® (lonapegsomatropin-tcgd) for the treatment of adults with growth hormone deficiency (GHD). Already approved in 2021 for pediatric GHD, SKYTROFA is a once-weekly prodrug of somatropin (human growth hormone), designed for sustained release of unmodified hGH, addressing the challenges of daily injection regimens.

“Our market research shows SKYTROFA is the treatment of choice for pediatric GHD among patients and physicians, and we are pleased to expand its availability in the United States for the treatment of adults initiating therapy or switching from another growth hormone therapy. This important milestone is the first of many planned label expansions supporting our goal to become the leading endocrinology rare disease company,” said Jan Mikkelsen, President and Chief Executive Officer at Ascendis Pharma. 

The approval was granted based on positive results from the Phase 3 foresiGHt trial, a randomized, double-blind, placebo- and active-controlled study. The trial evaluated the safety and efficacy of weekly TransCon hGH compared to daily somatropin and placebo in adults diagnosed with GHD. SKYTROFA demonstrated significant clinical benefits while maintaining a strong safety profile.

“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 disturbance, and reduced physical and mental drive. Despite this, adherence to daily somatropin injections that 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, 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., Barrow Neurological Institute, University of Arizona College of Medicine and Creighton University School of Medicine in Phoenix, Arizona. 

This marks a key milestone for Ascendis, expanding SKYTROFA’s label and reinforcing the company’s commitment to providing long-acting hormone replacement therapies. The once-weekly dosing option could improve treatment adherence and quality of life for adult patients living with this rare endocrine disorder.