SKYTROFA receives FDA approval for adult growth hormone deficiency

The U.S. Food and Drug Administration has approved SKYTROFA (lonapegsomatropin-tcgd) for treating adults with growth hormone deficiency, Ascendis Pharma A/S (NASDAQ: ASND) announced.

The once-weekly injection provides an alternative to daily somatropin treatments that have been the standard of care for more than 25 years. SKYTROFA was previously approved by the FDA in 2021 for pediatric growth hormone deficiency.

The approval was based on results from foresiGHt, a Phase 3 randomized clinical trial that compared weekly SKYTROFA with weekly placebo and daily somatropin in adults with growth hormone deficiency. The trial included both double-blind placebo-controlled and open-label active-controlled arms.

Growth hormone deficiency in adults results from decreased or total loss of growth hormone production and can lead to abnormal body composition, dyslipidemia, and insulin resistance.

"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," said Jan Mikkelsen, President and Chief Executive Officer at Ascendis Pharma.

The company plans to initiate trials for additional indications including idiopathic short stature, SHOX deficiency, Turner syndrome, and small for gestational age in the fourth quarter of 2025. Combination therapy trials in achondroplasia and hypochondroplasia are also planned for the same timeframe.

Common side effects in adults include swelling due to fluid build-up and low thyroid hormone levels, according to the prescribing information.

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