For the past 25 years, children and adults with growth hormone deficiency (GHD) have been treated with daily injections of recombinant human growth hormone (or hGH), whose amino acid sequence is identical to endogenous growth hormone. While dosing of daily hGH has been optimized for safety and efficacy, the burden of daily injections contributes to two out of three patients missing more than one injection per week. Poor adherence to daily hGH treatment is associated with sub-optimal outcomes.
TransCon hGH is in phase 3 development as a once-weekly therapy for children with GHD. We believe TransCon hGH has potential to be the best-in-class long-acting hGH therapy.
Ascendis Pharma is also developing an auto-injector to administer TransCon hGH. The proprietary auto-injector is being designed to deliver a single, low-volume injection via a small needle comparable to those used with daily hGH therapies. To avoid cold storage, we are developing TransCon hGH for storage at room temperature. Our auto-injector is also being designed for automatic data capture and will include key features enabling integration with a connected healthcare system.
TransCon hGH is currently being evaluated in a phase 3 clinical trial – the heiGHt Trial – comparing it to a daily hGH in pediatric GHD. Top-line data have been reported for the phase 3 heiGHt Trial, which compared TransCon hGH to a daily hGH in pediatric GHD. Based on these results and data from the ongoing fliGHt and enliGHten Trials, we expect to file a Biologics License Agreement (BLA) in the first half of 2020.
Growth Hormone Deficiency
Children with GHD are characterized by short stature, metabolic abnormalities, psychosocial challenges and poor quality of life. GHD in adults is associated with premature mortality, increased adiposity or fat mass, as well as psychiatric-cognitive, cardiovascular, muscular, metabolic and skeletal abnormalities.