Positive CHMP opinion for TransCon hGH for patients with pediatric growth hormone deficiency.

TransCon hGH is a long-acting once-weekly, transiently pegylated somatropin that in the body releases somatropin (also called human growth hormone, or hGH), indicated for growth failure in children and adolescents aged from 3 years up to 18 years due to insufficient endogenous growth hormone secretion (growth hormone deficiency, or GHD). The European Commission final decision on the Company’s Marketing Authorisation Application (MAA) for TransCon hGH is expected within 67 days after the positive opinion, or by the end of January 2022.

The CHMP opinion is based on results submitted in the MAA from the Company’s Phase 3III heiGHt, fliGHt and enliGHten trials, which collectively treated more than 300 pediatric patients diagnosed with GHD. In 2020, Ascendis Pharma received a positive opinion from the Paediatric Committee of the European Medicines Agency for its TransCon hGH Paediatric Investigation Plan, based on the non-clinical safety program as well as data from the three Phase III clinical trials included in the MAA.

Earlier this year, the FDA approved TransCon hGH – marketed in the U.S. under the brand name Skytrofa (lonapegsomatropin-tcgd) – for the treatment of pediatric patients one year and older who weigh at least 11.5 kg (25.4 lb.) and have growth failure due to inadequate secretion of endogenous growth hormone. Ascendis Pharma is also developing TransCon hGH in Japan, and separately in China through Visen Pharmaceuticals.