The FDA has recently approved Skytrofa as a treatment for children at least one year of age who are facing growth failure as a result of an inadequate secretion of the endogenous growth hormone. This approval is so notable because it is the very first FDA approved therapy that provides a sustained release of somatropin over a one week period. This reduces the treatment burden for patients.

Growth hormone deficiency (GHD) is a rare condition that leads to short stature and complications with the metabolic process. It results from insufficient production of the growth hormone by the pituitary gland.

Skytrofa is an injection needed once each week. The FDA approval also includes the auto-injector as well as the cartridges which can be stored without refrigeration for 6 months.

The approval of this therapy comes after the Phase 3 height trial.

Phase 3 Trial

The Phase 3 investigation compared Skytrofa to Genotropin in a total of 161 patients diagnosed with growth hormone deficiency who had not been previously treated.

The primary endpoint of the investigation was the AHV (annual height velocity) by 52 weeks of treatment.

The research team also monitored AEs, changes in height SDS, patient preference, patient satisfaction, and more.

The results of this trial demonstrated that by 52 weeks, annual height velocity was .9 centimeters/year for those in the Skytrofa group and 10.3 centimeters/year for those in the Genotropin group.

Looking Forward

Researchers are so excited about this new approval because it can significantly reduce the treatment burden for patients because Skytrofa only needs to be administered once a week.

It is expected that Skytrofa will be on the market soon. A number of resources will also be made available to patients including educational information on how to utilize the injectors.

You can read more about this new approval here.