FDA Approves SKYTROFA® (Lonapegsomatropin-tcgd) for the Once-Weekly Treatment of Adults with Growth Hormone Deficiency

Core Viewpoint - The FDA has approved SKYTROFA (lonapegsomatropin-tcgd) for the treatment of growth hormone deficiency (GHD) in adults, expanding its previous approval for pediatric use, which is expected to improve treatment adherence and patient outcomes [1][2][3]. Company Overview - Ascendis Pharma A/S is a global biopharmaceutical company focused on innovative therapies for rare diseases, utilizing its TransCon technology platform to address unmet medical needs [10][12]. Product Details - SKYTROFA is a prodrug of somatropin, administered once weekly, providing sustained release of active, unmodified somatropin, which is expected to enhance patient adherence compared to daily injections [1][3]. - The approval was based on the results from the foresiGHt Phase 3 clinical trial, which demonstrated the efficacy and safety of SKYTROFA compared to placebo and daily somatropin [2]. Market Position - SKYTROFA is already recognized as the treatment of choice for pediatric GHD, and the company aims to establish it as a leading option for adult patients as well [3][6]. - Ascendis Pharma plans to initiate additional clinical trials for various conditions, including ISS, SHOX deficiency, Turner syndrome, and SGA, in Q4 2025, as part of its strategy to expand its product offerings [6][13].