FDA approves Ascendis’ Skytrofa

The US Food & Drug Administration (FDA) has approved Danish biotech Ascendis Pharma’s (Nasdaq: ASND) 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 III 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.