Ascendis Pharma Advances Adult Growth Hormone Deficiency Treatment With TransCon hGH And Receives FDA Acceptance

Ascendis Pharma A/S announced that the U.S. FDA has accepted its supplemental Biologics License Application (sBLA) for TransCon hGH (lonapegsomatropin-tcgd), marketed as SKYTROFA® for pediatric growth hormone deficiency, to expand its use in adults with growth hormone deficiency (GHD). The FDA has set a target decision date under the Prescription Drug User Fee Act (PDUFA) for July 27, 2025.

Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer, said in a statement, “This marks another step towards achieving our objective to expand SKYTROFA’s label beyond pediatric GHD and expand its reach to address new groups of patients. Adult GHD is an undertreated condition associated with significant comorbidities and higher annual healthcare costs compared to the 5-10% of patients who receive treatment, indicative of the high unmet need.”

The sBLA is supported by data from the foresiGHt trial, a Phase 3 study that assessed the efficacy and safety of weekly TransCon hGH compared to weekly placebo and daily hGH in 259 adults aged 23 to 80 with GHD. Participants were randomized into three groups and dosed based on age and estrogen intake, ensuring similar weekly hGH exposure across treatments. Results at Week 38 showed that TransCon hGH significantly reduced trunk fat and increased total body lean mass when compared to placebo.

The treatment was generally well-tolerated, with no study drug-related discontinuations and a safety profile comparable to daily hGH. This submission marks a step forward in expanding treatment options for adult GHD patients.