FDA Approves Long-Acting Growth Hormone

Developed by Ascendis Pharma, this is the first therapy that delivers growth hormone over one week.

The FDA has approved Skytrofa (lonapegsomatropin-tcgd), developed by Ascendis Pharma to treat children one year and older who have growth failure. This is the first FDA approved product that delivers somatropin by sustained release over one week.

In growth hormone deficiency, the pituitary gland does not produce enough growth hormone, which is important not only for height but also for a child’s overall endocrine health and development.

The approval is based on results from the phase 3 heiGHt trial, a 52-week, global trial that compared the once-weekly therapy with daily somatropin (Genotropin) in 161 treatment-naïve children. No serious adverse events or discontinuations related to the therapy were reported.

The once-weekly therapy uses TransCon technology, which was developed by Ascendis. TransCon molecules have three components: an unmodified parent drug, an inert carrier that protects it, and a linker that temporarily binds the two. When bound, the carrier inactivates and shields the parent drug from clearance. Because the drug is unmodified, its original mode of action is expected to be maintained, according to Ascendis officials.

“This 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 Skyrofa demonstrated higher annualized height velocity at week 52 compared to somatropin,” Paul Thornton, M.B. B.Ch., MRCPI, a clinical investigator and pediatric endocrinologist in Fort Worth, Texas, said in a statement.

Skyrofa will be available in single-use, prefilled cartridges in nine dosage strengths, allowing. They are designed for use only with the Skrofa Auto-Injector and may be stored at room temperature for up to six months.