Ascendis Pharma Advances Weekly Skytrofa Trial in Pediatric Growth Disorders: What Investors Should Watch

Ascendis Pharma A/S (ASND) announced an update on their ongoing clinical study.

Ascendis Pharma A/S is running a late-stage global study to compare its once-weekly growth hormone therapy, lonapegsomatropin (Skytrofa), with standard once-daily somatropin in children and adolescents with short stature from several growth disorders. Officially, this Phase 3 basket trial aims to show that a weekly injection can safely match or improve growth outcomes versus daily shots in patients with Turner syndrome, SHOX deficiency, small for gestational age, and idiopathic short stature, a potentially important step to expand Skytrofa’s use beyond its current niche.

The study tests two treatment approaches. One group receives once-weekly lonapegsomatropin injections for two years. The other starts with daily somatropin injections for one year, then switches to once-weekly lonapegsomatropin for the second year. Both drugs are injectable growth hormone therapies aimed at improving height and growth in children with specific growth hormone–sufficient conditions.

The trial is an interventional Phase 3 study with patients randomly assigned to one of the two treatment paths. It follows a parallel-group setup, meaning both treatment strategies run at the same time in different patients. The study is open-label, so doctors and families know which treatment is given. The main goal is to evaluate how well the weekly drug supports growth and how safe it is compared with the daily standard of care.

The study is currently recruiting, with sites in the United States, several European countries, and South Korea. The trial was first submitted in October 2025, signaling when formal regulatory review of the protocol began. Primary completion will come once the key growth outcomes over the planned treatment period are collected, which will be the main data point investors watch for proof of efficacy. Final completion will follow after full safety and follow-up data are in. The last update to the listing on January 6, 2026, confirms that the trial plan and status remain current.

For investors, this update reinforces Ascendis Pharma’s push to broaden Skytrofa’s market into multiple pediatric growth conditions that are already treated with daily growth hormone. If results show comparable or better growth with a weekly injection, Ascendis could capture share from established daily products in the hands of larger players, including companies like Novo Nordisk and Pfizer. The basket design also allows Ascendis to pursue several niche indications at once, which can enhance the long-term revenue profile if approvals follow. Near term, the trial’s recruiting status and fresh update should support sentiment around Ascendis’ pipeline visibility and potential for label expansion, though binary data risk remains until top-line results are released.

The study is ongoing and recently updated, with full details available on the ClinicalTrials.gov portal under its listed identifier.

To learn more about ASND’s potential, visit the Ascendis Pharma A/S drug pipeline page.