European Medicine Agency’s CHMP Grants Positive Opinion to Long-Acting Growth Hormone for Patients with Pediatric Growth Hormone Deficiency

In November, the European Medicine Agency’s Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending the granting of a marketing authorization for Ascendis Pharma’s lonapegsomatropin (TransCon hGH), a long-acting once-weekly, transiently pegylated somatropin that in the body releases somatropin, 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 CHMP opinion is based on results submitted in the MAA from the Ascendis Pharma’s Phase 3 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 3 clinical trials included in the MAA.

Earlier this year, the U.S. Food & Drug Administration 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. 

“[The FDA] approval represents an important new choice for children with GHD and their families, who will now have a once-weekly treatment option. In the pivotal head-to-head clinical trial, once-weekly SKYTROFA demonstrated higher annualized height velocity at week 52 compared to somatropin,” says Society member Paul Thornton, MB, BCh, MRCPI, a clinical investigator and pediatric endocrinologist in Fort Worth, Texas. “This once-weekly treatment could reduce treatment burden and potentially replace the daily somatropin therapies, which have been the standard of care for over 30 years.”