FDA approved Ascendis Pharma’s Skytrofa for children one year and older who have growth failure from inadequate secretion of endogenous growth hormone.
FDA approved Ascendis Pharma’s Skytrofa.
The U.S.Food and Drug Administration (FDA) approved Ascendis Pharma’s Skytrofa (lonapegsomatropin-tcgd) for children one year and older who have growth failure from inadequate secretion of endogenous growth hormone (GH).
Skytrofa, a once-weekly injection, is the first FDA-approved drug that delivers somatropin by sustained release over one week. The approval includes the new Skytrofa Auto-Injector and cartridges, which families can store at room temperature for up to six months.
The approval was based on the Phase III height trial data that compared Skytrofa to daily injections of Pfizer’s Genotropin (somatropin) in 161 treatment-naïve children with GHD. The primary endpoint was annual height velocity (AHV) at 52 weeks. 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, PK/PD in patients less than three years of age, and preference for and satisfaction with Skytrofa.
At week 52, the difference in AHV was 0.9 centimeters per year for Skytrofa compared to 10.3 centimeters per year for Genotropin.
Denmark’s Ascendis may not have much time to have the only once-weekly treatment market to itself. Pfizer and Opko Health have a target action date in October for their once-weekly treatment called somatrogon.
Significance of Skytrofa
Dr. Paul Thornton, clinical investigator and pediatric endocrinologist in Fort Worth, Texas, 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 somatropin. 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.”
GHD is a severe orphan disease marked by short stature and complications with metabolism. In this condition, the pituitary gland does not produce enough growth hormone.
Skytrofa will be Ascendis’ first approved product. The company pegs the human growth hormone market at $4 billion globally, with the U.S. making up $1.3 billion. The childhood GHD market is estimated to be around $700 million.
The company expects to launch the drug soon in the U.S. along with a “suite” of patient support options. These include educating families on the use of the injectors.
“Skytrofa is the first product using our innovative TransCon technology platform that we have developed from the design phase through non-clinical and clinical development, manufacturing and device optimization, and out to the patients,’ said Jan M. Mikkelsen, president and chief executive officer of Ascendis. “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.”
Mary Andrews, chief executive officer and co-founder of the MAGIC Foundation, a leader in endocrine health, advocacy, education, and support, added, “It is wonderful that patients and their families now have the option of a once-weekly growth hormone therapy. GHD is often overlooked and undertreated in our children and managing it can be challenging for families. We are excited about this news as treating GHD is important, and children have a short time to grow.”
