Ascendis Pharma Seeks FDA Approval For New Growth Hormone Treatment For Adults

Ascendis Pharma A/S has announced the submission of a supplemental Biologics License Application (sBLA) to the U.S. Food and Drug Administration (FDA) for TransCon hGH. This treatment, known as SKYTROFA® (lonapegsomatropin-tcgd) in the United States for children with growth hormone deficiency, is now being proposed for adults with growth hormone deficiency (GHD).

The recent submission is supported by the findings from the foresiGHt study, a Phase 3 trial designed to assess the effectiveness and safety of TransCon hGH in adults with growth hormone deficiency (GHD). This randomized, double-blind, placebo-controlled study involved 259 adults aged 23 to 80, who were assigned in a 1:1:1 ratio to receive either TransCon hGH, a placebo, or daily hGH. Participants were given a target fixed dose of TransCon hGH or daily hGH, adjusted according to their age and oral estrogen use, ensuring comparable weekly hGH doses.

At Week 38, TransCon hGH showed superior results on its primary and key secondary efficacy measures. Patients treated with TransCon hGH experienced a statistically significant decrease in trunk fat and an increase in total body lean mass compared to those receiving the placebo. The treatment was found to be generally safe and well tolerated, with no participants discontinuing due to adverse effects, and its safety profile was similar to that of daily hGH.

Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer, stated “The sBLA submission for TransCon hGH for adult GHD supports our strategy to develop SKYTROFA into a blockbuster product through label expansion and build upon our value leadership position in the U.S. growth hormone market. 

He continued, “Growth hormone plays a vital role in human health at all ages, and, with research showing that the majority of adults living with growth hormone deficiency are not currently treated for this condition, we believe once weekly TransCon hGH could provide a new potential treatment option to address this unmet medical need.”