Global Commitment to Transforming Patient Lives: FDA to Review Ascendis’ Application for TransCon hGH
Ascendis Pharma A/S, a worldwide pharmaceutical leader harnessing the transformative potential of its TransCon technology, strives to emerge as a foremost and cohesive biopharmaceutical entity dedicated to having a substantial impact on patient healthcare outcomes. The US Food & Drug Administration (FDA) has agreed to inspect the supplemental Biologics License Application (sBLA) from Ascendis, aimed at addressing adult growth hormone deficiency (GHD) with TransCon hGH (lonapegsomatropin-tcgd, commonly known as Skytrofa for children’s GHD). The FDA has targeted July 27, 2025, as the Prescription Drug User Fee Act (PDUFA) goal date for this review.
“This development propels us further in our mission to widen Skytrofa’s approved uses beyond childhood GHD, thereby catering to additional patient demographics,” stated Jan Mikkelsen, the President and CEO of Ascendis Pharma. “Adult GHD is a condition frequently overlooked, resulting in various health complications and increased healthcare expenses, affecting only 5-10% of those who require treatment, reflecting the pressing need for intervention.”
The sBLA submission hinges on findings from the foresiGHt study—a phase 3 randomized, double-blind, placebo-controlled, and open-label active-controlled trial. This study assessed the efficacy and safety of weekly TransCon hGH versus weekly placebo and daily human growth hormone (hGH) in adults with GHD. Encompassing 259 adults aged 23 to 80 years, the study utilized random allocation (1:1:1) to determine the appropriate dose of TransCon hGH, placebo, or daily hGH based on age and hormone intake. The dosage mirrored the weekly equivalent for both TransCon hGH and daily hGH. TransCon hGH emerged superior in primary and key secondary endpoints at Week 38, with participants experiencing significant reductions in trunk fat and increases in total body lean mass compared to the placebo group. The trial confirmed that TransCon hGH was safe and well-tolerated, with no drug-related discontinuations and safety akin to daily hGH treatment.
Growth hormone deficiency significantly influences health from childhood through adulthood, facilitating normal growth in children and sustaining proper body composition and cardiometabolic health in adults. For adults, it enhances protein synthesis, promotes fat usage, builds muscle mass, and regulates glucose levels. Adult GHD, marked by insufficient growth hormone production, leads to central obesity, metabolic disorders, reduced bone density, abnormal lipid and cardiovascular markers, generalized fatigue, weakened muscle tone, and psychological issues such as cognitive dysfunction, social withdrawal, decreased motivation, and depression.