FDA Approves Weekly Therapy for Pediatric Patients with Growth Hormone Deficiency - Drug Information Update

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FDA Center for Drug Evaluation and Research Division of Drug Information

FDA Approves Weekly Therapy for Pediatric Patients with Growth Hormone Deficiency

FDA has approved Skytrofa (lonapegsomatropin-tcgd) injection to treat pediatric patients age one year and older who weigh at least 11.5 kg (25.4 pounds) and have short stature due to inadequate secretion of endogenous growth hormone. Skytrofa is a human growth hormone (hGH) approved for pediatric patients to take by under-the-skin injection weekly. Other available FDA-approved hGH formulations for pediatric patients with growth hormone deficiency must be administered daily.

Growth hormone deficiency is a disorder characterized by inadequate growth hormone production from the anterior pituitary gland, a small gland located at the base of the brain that produces several hormones. Children with this disorder can receive growth hormone to stimulate growth.

Skytrofa was studied in a 52-week trial that enrolled 161 pre-pubescent research participants (average age was 8.5 years) with growth hormone deficiency who had received no previous growth treatment. Participants were randomly assigned to receive either weekly injection of Skytrofa or daily injection of somatropin, an FDA-approved human growth hormone. Efficacy was determined by the increase in growth rate after 52 weeks of treatment. Weekly Skytrofa treatment for 52 weeks resulted in a growth rate increase of 11.2 cm (4.4 inches) per year and was not inferior to daily somatropin treatment.

The most common side effects of Skytrofa include viral infection, pyrexia (fever), cough, nausea and vomiting, hemorrhage (blood loss), abdominal (stomach) pain, arthralgia (joint pain), arthritis, and diarrhea. Patients with acute critical illness, allergies to somatropin or any Skytrofa excipients, active malignancy, certain types of diabetic retinopathy (eye-related diabetes complications), and Prader-Willi syndrome with other risk factors should not take Skytrofa. Children with closed epiphyses (end part of a long bone) should also not take the drug.

Patients taking Skytrofa are also at increased risk of neoplasms (abnormal tissue growth), intracranial hypertension (buildup of pressure around the brain), fluid retention, hypoadrenalism (underactive adrenal gland), hypothyroidism (underactive thyroid), slipped capital femoral epiphysis (a pediatric hip condition), progression of preexisting scoliosis, and pancreatitis (inflammation of the pancreas).

Skytrofa may interact with glucocorticoid (a type of steroid) treatment, oral estrogen, insulin, and other antihyperglycemic (blood sugar-reducing) agents.

FDA granted the approval to Ascendis Pharma.