For the past 30 years, children and adults with growth hormone deficiency (GHD) have been treated with daily injections of recombinant human growth hormone (or hGH), which has an amino acid sequence that 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 an investigational long-acting prodrug in phase 3 development as a once-weekly therapy for children with GHD. We believe TransCon hGH has the potential to be the best-in-class long-acting hGH therapy.
Ascendis Pharma has also developed a proprietary auto-injector to administer TransCon hGH. The investigational auto-injector is designed to deliver a single, low-volume injection via a small needle comparable to those used with daily hGH therapies. Our auto-injector is also being designed for automatic data capture and includes key features enabling integration with a connected healthcare system. Additionally, TransCon hGH can be stored at room temperature in a patient’s home.
In 2019, top-line data were reported for the phase 3 heiGHt Trial, which compared TransCon hGH to a daily hGH in pediatric GHD, and the phase 3 fliGHt or “switch” Trial, which evaluated TransCon hGH in those previously treated with commercially available daily hGH. Based on these results and data from the ongoing enliGHten Trial, we expect to file a Biologics License Application (BLA) with the U.S. Food and Drug Administration (FDA) in the first half of 2020, and a Marketing Authorisation Application (MAA) with the European Medicines Agency (EMA) in the second half of 2020.
TransCon hGH received Orphan Designation in pediatric GHD from the European Commission in 2019.
About 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.