Ascendis Pharma A/S announced that the U.S. Food & Drug Administration (FDA) has approved SKYTROFA (lonapegsomatropin-tcgd) for the treatment of adults with growth hormone deficiency (GHD). This approval marks a significant advancement for patients suffering from this rare condition characterized by decreased or absent growth hormone production.
SKYTROFA, previously approved by the FDA in 2021 for pediatric GHD, is a once-weekly prodrug of somatropin (human growth hormone) that offers sustained release of the active hormone. This new treatment option aims to improve adherence by reducing the burden of daily injections, which have been the standard for over 25 years.
The FDA’s decision was based on results from the Phase 3 foresiGHt trial, which demonstrated that weekly SKYTROFA was effective and safe compared to both placebo and daily somatropin injections in adults with GHD.
Dr. Kevin Yuen of the Barrow Neurological Institute highlighted the challenges faced by adults with GHD, including metabolic complications and impaired quality of life, and noted the potential benefits of a once-weekly treatment regimen in improving patient adherence and outcomes.
Jan Mikkelsen, President and CEO of Ascendis Pharma, emphasized that SKYTROFA is already the preferred treatment for pediatric GHD and expressed optimism about expanding its use to adult patients in the U.S. This approval is part of Ascendis Pharma’s broader strategy to become a leader in endocrinology rare diseases through ongoing label expansions.