Ascendis Pharma Secures FDA Approval For SKYTROFA To Treat Adult Growth Hormone Deficiency

• First of many planned label expansions supporting Vision 2030 goal to become the leading endocrinology rare disease company
• On track to initiate basket trial for ISS, SHOX deficiency, Turner syndrome, and SGA*, as well as combination therapy trials in achondroplasia and hypochondroplasia, in Q4 2025

COPENHAGEN, Denmark, July 28, 2025 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (NASDAQ:ASND) today announced that the U.S. Food & Drug Administration (FDA) has approved SKYTROFA® (lonapegsomatropin-tcgd; developed as TransCon hGH) for the replacement of endogenous growth hormone in adults with growth hormone deficiency (GHD), a rare disorder resulting from decreased or total loss of growth hormone production. Lonapegsomatropin (approved by the FDA in 2021 for the treatment of pediatric GHD) is a prodrug of somatropin (human growth hormone, or hGH) administered once weekly, providing sustained release of active, unmodified somatropin.

The FDA's approval of SKYTROFA for adult GHD was based on results from foresiGHt, a Phase 3 randomized, parallel-arm, placebo-controlled (double-blind) and active-controlled (open-label) clinical trial that compared the efficacy and safety of weekly TransCon hGH with weekly placebo and daily somatropin in adults with GHD.