The European Medicines Agency (EMA) has given the 'green light' to lonapegsomatropin (TransCon hGH, Ascendis Pharma), a long-acting, once-weekly formulation of human growth hormone, for growth failure in children and adolescents, aged 3 to 18 years, due to insufficient endogenous growth hormone secretion (growth hormone deficiency or GHD).
The EMA Committee for Medicinal Products for Human Use (CHMP) gave a positive opinion on lonapegsomatropin last week. Full marketing approval for all European Union countries normally follows a positive opinion within 67 days, or by the end of January 2022.
The drug molecule consists of a prodrug of somatropin that is inactive when bound to a proprietary TransCon (transient conjugation) inert carrier using a TransCon linker. The three-part molecule breaks apart after injection, exposing the active somatropin (also called human growth hormone) that is slowly released.
The CHMP opinion is based on results from Ascendis Pharma's Phase 3 heiGHt, fliGHt, and enliGHten trials, which collectively treated more than 300 pediatric patients diagnosed with GHD, according to the company.
"This milestone brings us a step closer to realizing our goal of being able to offer a once-weekly treatment of unmodified somatropin to patients in Europe," said Dana Pizzuti, MD, chief medical officer and senior vice president of development operations at Ascendis Pharma, in a company press statement.
Earlier this year, the US Food and Drug Administration approved TransCon hGH, marketed in the United States as Skytrofa (lonapegsomatropin-tcgd), for the treatment of pediatric patients age 1 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.
At the time, Bradley S. Miller, MD, PhD, told Medscape Medical News: "I am excited to be able to reduce the number of shots for some children requiring growth hormone therapy," with this new dosing option.
"I am hopeful that a once-weekly growth hormone...will improve adherence to growth hormone therapy leading to improved growth and metabolic outcomes," added Miller, professor and division director, pediatric endocrinology, at the University of Minnesota Masonic Children's Hospital in Minneapolis.
Ascendis Pharma is also developing TransCon hGH in Japan, and separately, in China through VISEN Pharmaceuticals.
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Cite this: EU Green Light for Once-Weekly Growth Hormone for Kids - Medscape - Nov 16, 2021.