Ascendis Pharma has submitted a supplemental biologics licence application (sBLA) to the US Food and Drug Administration (FDA) for TransCon hGH to treat adults with growth hormone deficiency (GHD).
TransCon hGH is marketed in the US as SKYTROFA for paediatric GHD.
The sBLA is supported by the results from the foresiGHt Phase III trial, which demonstrated the therapy’s efficacy and safety.
This study included 259 adults aged between 23 and 80 years with GHD. Participants were randomised in a 1:1:1 ratio and received either a target fixed dose of TransCon hGH, a placebo, or daily hGH.
The doses were titrated based on the participants’ age and oral estrogen intake, ensuring approximately equivalent hGH milligrams per week for both TransCon hGH and daily hGH.
At week 38 of the trial, TransCon hGH-treated patients reported a statistically significant reduction in trunk fat and an increase in total body lean mass compared to the placebo group, achieving superiority on its primary efficacy and key secondary efficacy endpoints.
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Ascendis Pharma president and CEO Jan Mikkelsen said: “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 US growth hormone market.
“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.”
Ascendis Pharma received FDA approval in August 2024 for YORVIPATH to treat hypoparathyroidism in adults.
