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Skytrofa (LONAPEGSOMATROPIN)
Skytrofa works by mimicking the natural growth hormone to stimulate growth in individuals with growth hormone deficiency.
Skytrofa (LONAPEGSOMATROPIN) is a recombinant human growth hormone developed by Ascendis Pharma Endocrinology Division A/S. It targets the growth hormone receptor and is used to treat growth failure due to inadequate secretion of endogenous growth hormone. Skytrofa is a small molecule modality and was FDA-approved in 2021. It is currently patented and owned by Ascendis Pharma Endocrinology Division A/S. Key safety considerations include the potential for injection site reactions and allergic reactions.
At a glance
| Generic name | LONAPEGSOMATROPIN |
|---|---|
| Sponsor | Ascendis Pharma Encocrinology Div A/S |
| Drug class | Recombinant Human Growth Hormone [EPC] |
| Target | Growth hormone receptor |
| Modality | Recombinant protein |
| Therapeutic area | Metabolic |
| Phase | FDA-approved |
| First approval | 2021 |
| Annual revenue | 446 |
Mechanism of action
SKYTROFA is pegylated human growth hormone (somatropin) for once-weekly subcutaneous injection [see Pharmacokinetics (12.3)].Somatropin binds to the growth hormone (GH) receptor in the cell membrane of target cells resulting in intracellular signal transduction and host of pharmacodynamic effects. Somatropin has direct tissue and metabolic effects, and indirect effects mediated by insulin-like growth factor-1 (IGF-1), including stimulation of chondrocyte differentiation and proliferation, stimulation of hepatic glucose output, protein synthesis and lipolysis. Somatropin stimulates skeletal growth in pediatric patients with growth hormone deficiency (GHD) as result of effects on the growth plates (epiphyses) of long bones.
Approved indications
- Growth failure due to inadequate secretion of endogenous growth hormone (GH)
Common side effects
- Infection, viral
- Pyrexia
- Cough
- Nausea and vomiting
- Hemorrhage
- Diarrhea
- Abdominal pain
- Arthralgia and arthritis
- Viral infection
Key clinical trials
- A Phase 3 Trial of the Safety, Tolerability and Efficacy of TransCon hGH Weekly Versus Daily hGH in Children With Growth Hormone Deficiency (GHD) (PHASE3)
- Trial Investigating the Efficacy and Safety of Weekly Lonapegsomatropin Compared to Daily Somatropin in Children and Adolescents With Short Stature or Growth Failure Due to Growth Hormone Sufficient Disorders (PHASE3)
- A Phase 2 Clinical Trial to Evaluate Efficacy, Safety, and Tolerability of Navepegritide in Combination With Lonapegsomatropin in Children With Achondroplasia (PHASE2)
- A US Non-interventional, Effectiveness and Safety Study of Patients Treated With SKYTROFA
- A Post-Authorisation Safety Study (PASS) of Patients Treated With Lonapegsomatropin
- A Trial to Investigate Different Doses of Lonapegsomatropin Compared to Somatropin in Individuals With Turner Syndrome (PHASE2)
- A Trial to Investigate Long Term Efficacy and Safety of Lonapegsomatropin in Adults With Growth Hormone Deficiency (PHASE3)
- A Trial to Compare the Efficacy and Safety of Once-weekly Lonapegsomatropin With Placebo and a Daily Somatropin Product in Adults With Growth Hormone Deficiency (PHASE3)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| FDA label | Mechanism, indications, dosing, boxed warnings, drug interactions |
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
| SEC EDGAR | Revenue + earnings |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Skytrofa CI brief — competitive landscape report
- Skytrofa updates RSS · CI watch RSS
- Ascendis Pharma Encocrinology Div A/S portfolio CI