European Union - English - EMA (European Medicines Agency)

ascendis pharma endocrinology division a/s - lonapegsomatropin - growth and development - pituitary and hypothalamic hormones and analogues - growth failure in children and adolescents aged from 3 years up to 18 years due to insufficient endogenous growth hormone secretion (growth hormone deficiency [ghd]),

United States - English - NLM (National Library of Medicine)

ascendis pharma endocrinology, inc. - lonapegsomatropin (unii: op35x9610y) (lonapegsomatropin - unii:op35x9610y) - skytrofa (lonapegsomatropin-tcgd) is a human growth hormone indicated for the treatment of pediatric patients 1 year and older who weigh at least 11.5 kg and have growth failure due to inadequate secretion of endogenous growth hormone (gh). skytrofa is contraindicated in patients with: - acute critical illness after open heart surgery, abdominal surgery or multiple accidental trauma, or those with acute respiratory failure due to the risk of increased mortality with use of pharmacologic doses of somatropin [see warnings and precautions (5.1)] . - hypersensitivity to somatropin or any of the excipients in skytrofa. severe systemic hypersensitivity reactions, including anaphylactic reactions and angioedema, have been reported [see warnings and precautions (5.2)]. - closed epiphyses. - active malignancy due to the risk of malignancy progression [see warnings and precautions (5.3)]. - active proliferative or severe non-proliferative diabetic retinopathy because treatment with somatropin may worsen this condition. - prader-willi syndrome who are severely obese, have a history of upper airway obstruction or sleep apnea or have severe respiratory impairment due to the risk of sudden death [see warnings and precautions (5.13)]. risk summary there are no available data on lonapegsomatropin-tcgd use in pregnant patients to evaluate a drug-associated risk of major birth defects, miscarriage or adverse maternal or fetal outcomes. available published data over several decades for somatropin, the active component of lonapegsomatropin-tcgd, have not identified a drug-associated risk of major birth defects, miscarriage or adverse maternal or fetal outcomes. in animal reproduction studies, there was no evidence of embryo-fetal or neonatal harm when pregnant rats were administered subcutaneous lonapegsomatropin-tcgd at doses up to 13-fold the clinical dose of 0.24 mg/kg/week (see data) . the estimated background risk of birth defects and miscarriages for the indicated population is unknown. all pregnancies have a background risk of birth defect, loss, or other adverse outcomes. in the u.s. general population, the estimated background risk of major birth defects and miscarriages in clinically recognized pregnancies is 2% to 4% and 15% to 20%, respectively. data animal data no embryonic or fetal development toxicities occurred in rats administered subcutaneous lonapegsomatropin-tcgd at doses up to 13-fold the clinical dose of 0.24 mg/kg/week. in a peri- and post-natal developmental study in rats, there were no adverse effects on the pregnant/lactating female or on development of the conceptus and the offspring following exposure of the female from implantation through weaning to doses of a structurally related pegylated somatropin prodrug up to 13-fold the clinical dose of 0.24 mg/kg/week. risk summary there are no data on the presence of lonapegsomatropin-tcgd in human milk, effects on the breastfed infant, or effects on milk production. high molecular weight therapeutic proteins, including lonapegsomatropin-tcgd, are expected to have low passage into human milk and limited systemic exposure in the breastfed infant. additionally, published data indicate that exogenous somatropin does not increase normal human milk concentrations of growth hormone. no adverse effects on the breastfed infant have been reported with somatropin. the developmental and health benefits of breastfeeding should be considered along with the mother's clinical need for skytrofa and any potential adverse effects on the breastfed infant from skytrofa or from the underlying maternal condition. safety and effectiveness of skytrofa have been established in pediatric patients 1 year and older and who weigh at least 11.5 kg. pediatric use was established in a controlled study of 161 treatment-naïve pediatric patients ages 3 to 13 years and by supportive data in pediatric patients 1 year and older [see adverse reactions (6) and clinical studies (14)] . the safety and effectiveness of skytrofa in children less than 1 year of age have not been established. use of somatropin in pediatric patients with prader-willi syndrome has been associated with reports of sudden death. skytrofa is not indicated for the treatment of pediatric patients with growth failure due to genetically confirmed prader-willi syndrome [see warnings and precautions (5.13)] . skytrofa is a prodrug of somatropin. somatropin is not a controlled substance. inappropriate use of somatropin may result in significant negative health consequences. somatropin is not associated with drug related withdrawal adverse reactions.