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Drug

Lonapegsomatropin

Approved · EMA
Emerging researchLate-stage development

Also known as Skytrofa, TransCon hGH.

RxNorm2569562UNIIOP35X9610Y
1
Research papers
1
Active clinical trials
1
Regulatory events
Latest activity
beta

Recent clinical, regulatory, research and industry developments relating to this drug.

A Multicenter, Open-Label, Extension Trial to Investigate Long Term Efficacy and Safety of Lonapegsomatropin in Adults With Growth Hormone Deficiency

Clinical trial2026-07-07Results posted · ClinicalTrials.gov

enliGHten: A Multicenter, Phase 3, Long-Term, Open-Label Trial Investigating Safety and Efficacy of TransCon hGH Administered Once-Weekly in Children With Growth Hormone Deficiency (GHD) Who Have Completed a Prior TransCon hGH Clinical Trial

Clinical trial2026-07-07Results posted · ClinicalTrials.gov

fliGHt: A Multicenter, Phase 3, Open-Label, 26-Week Trial Investigating the Safety, Tolerability and Efficacy of TransCon hGH Administered Once Weekly in Children With GHD

Clinical trial2026-07-07Results posted · ClinicalTrials.gov

foresiGHt: A Multicenter, Randomized, Parallel-arm, Placebo-controlled (Double- Blind) and Active-controlled (Open-label) Trial to Compare the Efficacy and Safety of Once-weekly Lonapegsomatropin With Placebo and a Daily Somatropin Product in Adults With Growth Hormone Deficiency

Clinical trial2026-07-07Results posted · ClinicalTrials.gov

New InsiGHTS: A Multicenter, Phase 2, Randomized, Open-label, Active-controlled, Parallel Group Clinical Trial to Investigate the Safety, Tolerability, and Efficacy of Different Dose Levels of Once-weekly Lonapegsomatropin Compared to Daily Somatropin in Prepubertal Individuals With Turner Syndrome

Clinical trial2024-10-18Primary completion · ClinicalTrials.gov

Safety and Efficacy of Lonapegsomatropin in Children With Growth Hormone Deficiency: enliGHten Trial 2-Year Results.

Research2022-06-01The Journal of clinical endocrinology and metabolism

The Efficacy, Safety and Tolerability of TransCon hGH Administered Weekly Versus Daily hGH in Prepubertal Children With Growth Hormone Deficiency: a Randomized, Open-lable, Active-controlled, Parallel-group Study in China

Clinical trial2022-04-01Primary completion · ClinicalTrials.gov

Approval: Skytrofa (previously Lonapegsomatropin Ascendis Pharma) (EMA)

Regulatory2022-01-11EMA

Profile

Identifiers & mechanism

Canonical identifiers, marketed brand names and mechanism, resolved across RxNorm, ChEMBL and ATC.

Canonical name
Lonapegsomatropin
Aliases & brands
SkytrofaTransCon hGH
RxNorm CUI
2569562
UNII
OP35X9610Y
Regulatory jurisdictions
ema

Regulatory timeline

1 event

The complete regulatory record, grouped by authority — approvals, safety advisories and label changes. Each authority shows its most recent events; expand one to read its full history.

Earliest approval
2022-01-11
Latest approval
2022-01-11
Authorities
EMA
Total events
1
emaEuropean Medicines Agency· 1 event
2022-01-11Approval
Approval: Skytrofa (previously Lonapegsomatropin Ascendis Pharma) (EMA)

Indication: Growth failure in children and adolescents aged from 3 years up to 18 years due to insufficient endogenous growth hormone secretion (growth hormone deficiency [GHD])

Evidence ↗

Contains information from the European Medicines Agency (European Medicines Agency), © EMA, reused under CC BY 4.0.

Clinical trials

6 trials

The current development programme across all trial phases — status mix, phase distribution and the late-stage studies shaping the evidence base.

Development programme
CLINICALTRIALS.GOV · LIVE REGISTRY
6
registered trials across all phases
LATEST COMPLETION 2024
1
Active studies
5
Late-stage (III+)
4
Completed
1
Discontinued
PHASE DISTRIBUTIONn = 6
Phase 21Phase 35

Late-stage studies

Phase III+ trials still open or recently active — where late-stage evidence is being generated.

A Multicenter, Open-Label, Extension Trial to Investigate Long Term Efficacy and Safety of Lonapegsomatropin in Adults With Growth Hormone Deficiency

CompletedPH IIIAscendis Pharma Endocrinology Division A/Sn=2202024

foresiGHt: A Multicenter, Randomized, Parallel-arm, Placebo-controlled (Double- Blind) and Active-controlled (Open-label) Trial to Compare the Efficacy and Safety of Once-weekly Lonapegsomatropin With Placebo and a Daily Somatropin Product in Adults With Growth Hormone Deficiency

CompletedPH IIIAscendis Pharma Endocrinology Division A/Sn=2642023

enliGHten: A Multicenter, Phase 3, Long-Term, Open-Label Trial Investigating Safety and Efficacy of TransCon hGH Administered Once-Weekly in Children With Growth Hormone Deficiency (GHD) Who Have Completed a Prior TransCon hGH Clinical Trial

CompletedPH IIIAscendis Pharma A/Sn=2982023

The Efficacy, Safety and Tolerability of TransCon hGH Administered Weekly Versus Daily hGH in Prepubertal Children With Growth Hormone Deficiency: a Randomized, Open-lable, Active-controlled, Parallel-group Study in China

Unknown statusPH IIIVisen Pharmaceuticals (Shanghai) Co., Ltd.n=1502022

fliGHt: A Multicenter, Phase 3, Open-Label, 26-Week Trial Investigating the Safety, Tolerability and Efficacy of TransCon hGH Administered Once Weekly in Children With GHD

CompletedPH IIIAscendis Pharma Endocrinology Division A/Sn=1462019

Recent completions

Trials that read out recently, adding to the completed evidence base.

A Multicenter, Open-Label, Extension Trial to Investigate Long Term Efficacy and Safety of Lonapegsomatropin in Adults With Growth Hormone Deficiency

CompletedPH IIIAscendis Pharma Endocrinology Division A/Sn=2202024

foresiGHt: A Multicenter, Randomized, Parallel-arm, Placebo-controlled (Double- Blind) and Active-controlled (Open-label) Trial to Compare the Efficacy and Safety of Once-weekly Lonapegsomatropin With Placebo and a Daily Somatropin Product in Adults With Growth Hormone Deficiency

CompletedPH IIIAscendis Pharma Endocrinology Division A/Sn=2642023

enliGHten: A Multicenter, Phase 3, Long-Term, Open-Label Trial Investigating Safety and Efficacy of TransCon hGH Administered Once-Weekly in Children With Growth Hormone Deficiency (GHD) Who Have Completed a Prior TransCon hGH Clinical Trial

CompletedPH IIIAscendis Pharma A/Sn=2982023

fliGHt: A Multicenter, Phase 3, Open-Label, 26-Week Trial Investigating the Safety, Tolerability and Efficacy of TransCon hGH Administered Once Weekly in Children With GHD

CompletedPH IIIAscendis Pharma Endocrinology Division A/Sn=1462019

Research activity

1 papers

Key research shaping understanding of this drug, combining the latest publications with the most influential evidence.

Most influential

Safety and Efficacy of Lonapegsomatropin in Children With Growth Hormone Deficiency: enliGHten Trial 2-Year Results.

The Journal of clinical endocrinology and metabolism · 2022 · 17 cites
Recent papers

Safety and Efficacy of Lonapegsomatropin in Children With Growth Hormone Deficiency: enliGHten Trial 2-Year Results.

The Journal of clinical endocrinology and metabolism · 2022 · 17 cites
Major research themes2
Dwarfism, Pituitary1Human Growth Hormone1
Journals, researchers & institutions
Top journals
  • The Journal of clinical endocrinology and metabolism1
Leading researchers
  • Aghajanova EM1
  • Beckert M1
  • Cappa M1
  • Casella SJ1
  • Chaychenko T1
  • Chertock ED1
  • Chessler SD1
  • Hofman PL1
Leading institutions
free-text, unnormalised
  • Bashkir State Medical University1
  • Center of Excellence in Diabetes and Endocrinology1
  • Children's Hospital at Dartmouth-Hitchcock1
  • Children's Hospital. P. A. Kyriakou1
  • Liggins Institute1
  • MHI Regional Child Clinical Hospital1
References & data sources
  • RxNorm (U.S. National Library of Medicine) — drug identity
  • Europe PMC — research literature
  • ClinicalTrials.gov — clinical trials
  • Regulatory event sources are credited in the Regulatory Timeline above.