FDA Accepts Ascendis Pharma’s Supplemental Biologics License Application for TransCon™ hGH for the Treatment of Adults with Growth Hormone Deficiency

Core Viewpoint - Ascendis Pharma A/S has received FDA acceptance for the review of its supplemental Biologics License Application for TransCon hGH in adult growth hormone deficiency, with a PDUFA goal date set for July 27, 2025 [1][2]. Company Overview - Ascendis Pharma is focused on developing innovative therapies using its TransCon technology platform, aiming to make a significant impact on patients' lives [4]. Product Development - The sBLA submission is based on the Phase 3 foresiGHt trial, which demonstrated the efficacy and safety of TransCon hGH compared to placebo and daily human growth hormone in adults with GHD [2]. - The trial involved 259 adults aged 23 to 80, showing that TransCon hGH led to a significant reduction in trunk fat and an increase in total body lean mass at Week 38 compared to placebo [2]. Market Need - Adult growth hormone deficiency is significantly undertreated, with only 5-10% of patients receiving treatment, indicating a high unmet medical need [2][3]. - Symptoms of adult GHD include central obesity, metabolic syndrome, decreased bone density, and psychological issues, highlighting the importance of effective treatment options [3].