Early FDA approval for Ascendis Pharma's Skytrofa for pediatric GHD

A month ahead of the expected decision date, the US Food and Drug Administration yesterday approved Skytrofa (lonapegsomatropin-tcgd) for the treatment of pediatric patients one 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 (GH), says Danish biotech Ascendis Pharma (Nasdaq: ASND).

As a once-weekly injection, Skytrofa is the first FDA approved product that delivers somatropin (growth hormone) by sustained release over one week.

“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 somatropin,” said Paul Thornton, a clinical investigator and pediatric endocrinologist in Fort Worth, Texas. “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,” he noted.