FDA Approves Skytrofa for Pediatric Patients with Growth Hormone Deficiency

Today, the Food and Drug Administration (FDA) has approved the human growth hormone Skytrofa for pediatric patients with growth hormone deficiency who are 1 year or older with a weight of at least 25.4 pounds and short stature due to inadequate secretion of endogenous growth hormone.

The injection is intended to be taken through an under-the-skin weekly injection, while other FDA-approved hGH formulations for pediatric patients with growth hormone deficiencies must be taken daily.

The hormone was studied in a recent 52-week trial that enrolled 161 participants who had received no previous growth treatment. Participants were randomly assigned to receive the weekly injection or a daily injection of another FDA-approved hormone somatropin.

Weekly Skytrofa treatment resulted in a growth rate increase of 11.2 cm (4.4 inches) per year and was not inferior to daily somatropin treatment.

The FDA granted approval to Ascendis Pharma, though side effects and risks of Skytrofa are evident. The most common side effects of Skytrofa include viral infection, fever, cough, nauseas, arthritis, and more.

Additionally, patients taking Skytrofa are at an increased risk of neoplasms, intracranial hypertension, fluid retention, hypoadrenalism, hypothyroidism, slipped capital femoral epiphysis, progression of preexisting scoliosis, and pancreatitis.