Ascendis Pharma A/S (NASDAQ: ASND) has approval for SKYTROFA (lonapegsomatropinc-tcgd) in pediatric patient treatment for those above one year and weighing 11.5 Kg that has growth failure because of inadequate endogenous growth hormone secretion.
SKYTROFA is the first FDA approved product offering somatropin
SKYTROFA is the first FDA-approved medicine to provide somatropin (growth hormone) over a one-week period as a once-weekly injection.
Clinical investigator Paul Thornton said, “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. 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.”
Metabolic issues and short stature are symptoms of growth hormone insufficiency, a devastating orphan illness. The pituitary gland does not create enough growth hormone in GHD, which is critical for a child’s general endocrine development, and health not just for height.
SKYTROFA can be stored at room temperature for six months
The new SKYTROFA cartridges and Auto-Injector have received FDA approval, allowing families to store the drug at room temperature for around six months after first removing it from the refrigerator. Patients who move from daily injections to weekly injections can have up to 86% fewer injection days per year.
Ascendis CEO Jan Mikkelsen said, “SKYTROFA is the first product using our innovative TransCon technology platform that we have developed from design phase through non-clinical and clinical development, manufacturing and device optimization, and out to the patients. It reflects our commitment and dedication to addressing unmet medical needs by developing a pipeline of highly differentiated proprietary products across multiple therapeutic areas. We are grateful to the patients, caregivers, clinicians, clinical investigators, and our employees, who have all contributed to bringing this new treatment option to children in the U.S. with GHD.”
