At a glance
- Lonapegsomatropin is an FDA-approved drug, marketed as Skytrofa (first approval: August 25, 2021).
- PubMed lists 47 publications mentioning it, including 3 randomized controlled trials and 0 animal-only studies.
- ClinicalTrials.gov lists 12 registered studies testing it (5 active).
- FDA's adverse event system (FAERS) holds 626 reports mentioning it; 60 classified as serious. Reports do not prove causation.
Research topics
Most frequent MeSH subject headings in the latest PubMed records (English, as indexed).
Regulatory status
Drugs@FDA applications
| Application | Product | Status | First approval | Sponsor |
|---|---|---|---|---|
| BLA761177 | SKYTROFA | Approved, marketed | August 25, 2021 | ASCENDIS PHARMA ENCOCRINOLOGY DIV A/S |
Source: Drugs@FDA (openFDA) · verified October 30, 2026 · API query
FDA compounding safety list
Not on FDA's list of bulk drug substances that may present significant safety risks in compounding.
Source: FDA.gov — Category 2 bulk drug substances · verified October 30, 2026
Removal of a substance from a regulatory list does not mean it has been approved. A "research use only" label does not make a product safe or legal for human use.
Scientific publications
Counts are PubMed records that mention the peptide in the title or abstract. Categories overlap, and a mention is not the same as testing the peptide as a treatment. Each number opens the exact PubMed search. Effects seen in laboratory or animal studies may not occur in humans. A registered clinical trial does not show that a substance is effective or safe.
Latest publications
- Efficacy and Safety of Lonapegsomatropin in Children With Turner Syndrome: 52-Week Results From the New InsiGHTS Trial.
- Long-term Safety and Efficacy of Once-Weekly Lonapegsomatropin in Adults With Growth Hormone Deficiency: Results From a 52-Week Open-Label Extension Trial.
- Correction to "Efficacy and Safety of Once-Weekly Lonapegsomatropin in Adults With Growth Hormone Deficiency: foresiGHt Trial Results".
- Immunogenicity Profile of Lonapegsomatropin During Long-Term Treatment in Children With Growth Hormone Deficiency.
- Treatment of growth hormone deficiency with long-acting growth hormones in children, adolescents and adults: Clinical evidence and practical considerations.
- Long-Acting Growth Hormone Versus Daily Growth Hormone for Growth Hormone Deficiency Patients: A Network Meta-Analysis of Clinical Trials.
- Diabetic ketoacidosis induced by long-acting growth hormone in a child without preexisting diabetes.
- Comparison of efficacy and safety between long-acting growth hormone and short-acting growth hormone in isolated growth hormone deficiency: a systematic review and meta-analysis of 16 randomized controlled trials involving 2,435 pediatric patients.
- Patient-Centric Design of the Lonapegsomatropin Auto-Injector for Growth Hormone Deficiency: Usability Validation of Safe and Effective Use.
- Navepegritide combined with lonapegsomatropin for the treatment of children with achondroplasia: 52-week results from the phase 2 COACH trial.
Source: PubMed (NCBI) · verified October 30, 2026
Clinical trials
- Registered
- 12
- Active
- 5
- With posted results
- 6
Conditions studied
Show 2 more studies
| Study | Status | Phase | Start |
|---|---|---|---|
| A Phase 3 Trial of the Safety, Tolerability and Efficacy of TransCon hGH Weekly Versus Daily hGH in Children With Growth Hormone Deficiency (GHD) | Completed | Phase 3 | December 13, 2016 |
| A Safety, Pharmacokinetic and Pharmacodynamic Study of ACP-001 (TransCon hGH) in Adults With Growth Hormone Deficiency | Completed | Phase 2 | Nov 2010 |
All 12 studies on ClinicalTrials.gov ↗
Source: ClinicalTrials.gov · verified October 30, 2026
Adverse event reports
- Reports
- 626
- Serious
- 60
- With death outcome
- 0
Most reported reactions
Adverse event reports do not prove that the substance caused these effects. Side effects not listed here may still occur. Reaction terms are MedDRA terms as reported to FAERS.
Source: FDA FAERS (FAERS data as of July 30, 2026) · verified October 30, 2026 · API query
Change history
No changes since monitoring began on October 30, 2026.