Ascendis Pharma Seeks FDA Approval for TransCon hGH in Adult Growth Hormone Deficiency
核心洞察
Ascendis Pharma has submitted a supplemental Biologics License Application (sBLA) to the FDA for TransCon hGH to treat adults with growth hormone deficiency (搜索) (GHD).
The sBLA is based on positive results from the Phase 3 foresiGHt trial, which demonstrated TransCon hGH's efficacy and safety in adults with GHD.
TransCon hGH showed statistically significant reductions in trunk fat and increases in lean body mass compared to placebo in the clinical trial.
Ascendis Pharma has announced the submission of a supplemental Biologics License Application (sBLA) to the U.S. Food and Drug Administration (FDA) for TransCon hGH (lonapegsomatropin-tcgd) for the treatment of adults with growth hormone deficiency (搜索) (GHD). This submission aims to expand the label of SKYTROFA, currently approved for pediatric GHD, to include adult patients. The potential approval could address a significant unmet need, as research indicates that a majority of adults with GHD are not currently receiving treatment.
The sBLA is supported by 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 once-weekly TransCon hGH with both weekly placebo and daily hGH in 259 adults with GHD, aged 23 to 80 years. Participants were randomized 1:1:1 to receive TransCon hGH, placebo, or daily hGH, with doses titrated based on age and estrogen intake to ensure equivalent hGH exposure per week between the TransCon hGH and daily hGH groups.
Efficacy and Safety Results
The foresiGHt trial demonstrated that TransCon hGH was superior to placebo on both primary and key secondary efficacy endpoints at Week 38. Patients treated with TransCon hGH experienced a statistically significant reduction in trunk fat and an increase in total body lean mass compared to those receiving placebo. The safety profile of TransCon hGH was comparable to that of daily hGH, with no discontinuations related to the study drug.
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, fatigue, and cognitive impairment. Growth hormone plays a crucial role in maintaining body composition, cardiometabolic health, and overall well-being in adults. Jan Mikkelsen, Ascendis Pharma’s President and CEO, stated that TransCon hGH could provide a new potential treatment option to address this unmet medical need.
TransCon Technology
TransCon hGH utilizes Ascendis Pharma's TransCon technology, designed to create new therapies that potentially offer best-in-class profiles. Ascendis Pharma is focused on applying this technology to develop treatments that can make a meaningful difference in patients’ lives.