FDA Accepts Ascendis Pharma's TransCon hGH Application for Adult Growth Hormone Deficiency
Key Insights
The FDA has accepted Ascendis Pharma's sBLA for TransCon hGH to treat adult growth hormone deficiency (search), setting a PDUFA date for July 27, 2025.
Phase 3 foresiGHt trial data demonstrated TransCon hGH's superiority in reducing trunk fat and increasing lean body mass compared to placebo.
TransCon hGH showed a strong safety profile, with tolerability comparable to daily hGH treatment and no discontinuations related to the study drug.
Ascendis Pharma A/S (Nasdaq: ASND) announced that the U.S. Food & Drug Administration (FDA) has accepted for review its supplemental Biologics License Application (sBLA) for TransCon hGH (lonapegsomatropin-tcgd), also known as SKYTROFA®, for adult growth hormone deficiency (search) (GHD). The FDA has set a Prescription Drug User Fee Act (PDUFA) goal date of July 27, 2025.
The sBLA submission is based on data from the Phase 3 foresiGHt trial, a randomized, parallel-arm, placebo-controlled (double-blind) and active-controlled (open-label) study. The trial compared the efficacy and safety of weekly TransCon hGH with weekly placebo and daily human growth hormone (hGH) in adults with GHD.
ForesiGHt Trial Results
The foresiGHt trial involved 259 adults with GHD, aged 23 to 80 years, who were randomized 1:1:1 to receive a target fixed dose of TransCon hGH, placebo, or daily hGH. The doses were titrated based on age and oral estrogen intake, ensuring approximately equivalent hGH mg/week for TransCon hGH and daily hGH.
TransCon hGH demonstrated superiority on its primary efficacy and key secondary efficacy endpoints at Week 38. Participants treated with TransCon hGH showed a statistically significant reduction from baseline in trunk fat and an increase in total body lean mass compared to placebo.
Safety and Tolerability
In the trial, TransCon hGH was generally safe and well-tolerated. There were no discontinuations related to the study drug, and the safety and tolerability were comparable to daily hGH treatment.
Addressing Unmet Needs in Adult GHD
Adult GHD is characterized by insufficient growth hormone production, leading to symptoms such as central obesity, metabolic syndrome, decreased bone density, and psychological symptoms like cognitive impairment and depression. According to Ascendis Pharma, only 5-10% of adult GHD patients currently receive treatment, highlighting a significant unmet medical need.
Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer, stated, "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."
Potential Impact
If approved, TransCon hGH could offer a new treatment option for adults with GHD, potentially improving body composition, cardiometabolic health, and overall well-being. The weekly administration of TransCon hGH may also improve treatment compliance compared to daily injections, leading to better patient outcomes.