FDA approves Skytrofa for pediatric growth hormone deficiency.

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

The approval includes the new Skytrofa Auto-Injector and cartridges which, after first removed from a refrigerator, allow families to store the medicine at room temperature for up to six months. With a weekly injection, patients switching from injections every day can experience up to 86 percent fewer injection days per year.

The FDA approval of Skytrofa was based on results from the phase III heiGHt Trial, a 52-week, global, randomized, open-label, active-controlled, parallel-group trial that compared once-weekly Skytrofa to daily somatropin (Genotropin) in 161 treatment-naïve children with GHDii. The primary endpoint was, AHV at 52 weeks for weekly Skytrofa and daily hGH treatment groups. Other endpoints included adverse events, injection-site reactions, incidence of anti-hGH antibodies, annualized height velocity, change in height SDS, proportion of subjects with IGF-1 SDS (0.0 to +2.0), PK/PD in subjects < 3 years, and preference for and satisfaction with Skytrofa.

At week 52, the treatment difference in AHV was 0.9 cm/year (11.2 cm/year for Skytrofa compared with 10.3 cm/year for daily somatropin) with a 95 percent confidence interval [0.2, 1.5] cm/year. The primary objective of non-inferiority in AHV was met for Skytrofa in this trial and further demonstrated a higher AHV at week 52 for lonapegsomatropin compared to daily somatropin, with similar safety, in treatment-naïve children with GHD. No serious adverse events or discontinuations related to Skytrofa were reported.