TransCon hGH is designed to release somatropin with the same mode of action and distribution as once-daily somatropin products, but with a once-weekly injection.1 The BLA for pediatric GHD is supported by the results a clinical development program that included eight clinical trials evaluating safety and efficacy in more than 400 subjects with GHD.
TransCon hGH is an investigational therapy that is not approved for use in any country. There is no long-acting growth hormone treatment approved by the FDA for the treatment of pediatric GHD. TransCon hGH has received orphan designation in both the U.S. and Europe as a treatment for GHD. The company plans to submit a Marketing Authorisation Application for TransCon hGH in pediatric GHD to the European Medicines Agency too in next few days.
Pediatric GHD is a serious orphan disease caused when the pituitary gland does not produce enough growth hormone. Children with GHD are not only characterized by short stature, but they also may experience metabolic abnormalities, psychosocial challenges and poor quality of life. For decades, the standard of care for GHD has been a daily subcutaneous injection of hGH, which improves growth and overall endocrine health.
TransCon refers to “transient conjugation.” The proprietary TransCon platform is an innovative technology designed to create new therapies that optimize therapeutic effect, including efficacy, safety and dosing frequency. TransCon molecules have three components: an unmodified parent drug, an inert carrier that protects it, and a linker that temporarily binds the two. When bound, the carrier inactivates and shields the parent drug from clearance. When injected into the body, physiologic conditions (e.g., pH and temperature) initiate the release of the active, unmodified parent drug in a predictable manner. Because the parent drug is unmodified, its original mode of action may be maintained. TransCon technology is designed to be applied broadly to a protein, peptide or small molecule in multiple therapeutic areas, and to be used systemically or locally.