FDA Alerts
Growth Hormone Disorders

FDA approves first once-weekly treatment for pediatric growth hormone deficiency

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The U.S. Food and Drug Administration has approved lonapegsomatropin (Skytrofa, Ascendis Pharma) for the treatment of pediatric patients with growth failure due to inadequate secretion of endogenous growth hormone, according to a press release.

This is the first weekly subcutaneous injectable growth hormone for children with growth hormone deficiency (GHD). Treatment is indicated for patients ≥1 year who weigh at least 25.4 lbs.

Approval was based on the results of the phase 3 heiGHt Trial that compared lonapegsomatropin to daily somatropin in 161 treatment-naïve children with GHD.

At week 52, AHV was 11.2 cm/year in patients treated with lonapegsomatropin compared with 10.3 cm/year for those treated with daily somatropin.

No serious adverse events related to lonapegsomatropin were reported.

“Today’s 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 somatropini,” said Paul Thornton, MB, BCh, MRCPI, a clinical investigator and pediatric endocrinologist in Fort Worth, Texas in a press release. “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.”

Read the full press release here.