Ascendis Pharma A/S announced that the U.S. Food & Drug Administration (FDA) has approved SKYTROFA (lonapegsomatropin-tcgd) for the once-weekly treatment of adults with growth hormone deficiency (GHD). This approval marks a significant label expansion for SKYTROFA, which was previously approved for pediatric GHD in 2021.
The FDA's decision was based on results from the Phase 3 foresiGHt trial, which compared the efficacy and safety of weekly TransCon hGH with weekly placebo and daily somatropin in adults with GHD. The trial demonstrated SKYTROFA's superiority on its primary and key secondary efficacy endpoints at Week 38, including a reduction in trunk fat and an increase in total body lean mass compared to placebo.
SKYTROFA's once-weekly injection and sustained release of unmodified somatropin are expected to improve real-world adherence and overall outcomes for adult GHD patients, an undertreated condition associated with significant comorbidities. This milestone is the first of many planned label expansions, supporting Ascendis Pharma's goal to become a leading endocrinology rare disease company.
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