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Lonapegsomatropin

Phase 3

Turner Syndrome | Monoclonal antibody | Other |Ascendis Pharma A/S|Last Updated: May 29, 2026

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedACTIVE_CONTROLLEDBiomarker
Total Trials2
Total Enrollment234
FDA Designations
No designations recorded
Clinical Trials (2)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT07221851Trial 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 DisordersPHASE3 RECRUITING 186Dec 12, 2025Mar 1, 2029May 29, 202615 United States, France +3
NCT05690386A Trial to Investigate Different Doses of Lonapegsomatropin Compared to Somatropin in Individuals With Turner SyndromePHASE2 ACTIVE NOT_RECRUITING 48Feb 15, 2023Dec 1, 2027May 13, 202619 United States
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Study Endpoints
Primary Endpoints
Annualized Height Velocity (AHV) (cm/year)
52 Weeks

To evaluate the efficacy of lonapegsomatropin as compared to somatropin in children and adolescents with TS, SHOX-D, SGA, or ISS

Secondary Endpoints
Annualized Height Velocity (AHV) (cm/year)
104 Weeks
Change from baseline in height standard deviation score (SDS)
52 Weeks and 104 Weeks
Change from baseline in Bone Age (years)
52 Weeks and 104 Weeks
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Study Design & Arms
AllocationRANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Lonapegsomatropin, once dailyEXPERIMENTALParticipants will receive Lonapegsomatropin by subcutaneous injection for 2 years (104 weeks)
somatropin, once dailyACTIVE_COMPARATORParticipants will receive somatropin by subcutaneous injection for 1 year (52 weeks) followed by lonapegsomatropin for 1 year (52 weeks)
Lonapegsomatropin at 0.24 mg hGH/kg/weekEXPERIMENTALLonapegsomatropin at 0.24 mg hGH/kg/week administered once-weekly by subcutaneous injection
Lonapegsomatropin at 0.30 mg hGH/kg/weekEXPERIMENTALLonapegsomatropin at 0.30 mg hGH/kg/week administered once-weekly by subcutaneous injection
Lonapegsomatropin at 0.36 mg hGH/kg/weekEXPERIMENTALLonapegsomatropin at 0.36 mg hGH/kg/week administered once-weekly by subcutaneous injection
Somatropin at 0.05 mg/kg/dayACTIVE_COMPARATORSomatropin at 0.05 mg/kg/day administered once-daily by subcutaneous injection
Interventions
NameTypeDescription
Lonapegsomatropin [SKYTROFA®]COMBINATION_PRODUCTSubcutaneous injection once weekly
Somatropin Pen InjectorCOMBINATION_PRODUCTSubcutaneous injection once daily
LonapegsomatropinBIOLOGICALOnce-weekly subcutaneous injection of Lonapegsomatropin
SomatropinDRUGOnce-daily subcutaneous injection of Somatropin
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Eligibility Criteria
Age Range2 Years — 17 Years
SexALL
Healthy VolunteersNo
Study Sites15

Inclusion Criteria: 1. Chronological age between ≥2 and \<18 years, at start of screening. 2. Naïve to growth hormone and growth hormone promoting therapies. 3. Prepubertal. 4. Able to stand without assistance. 5. Diagnosis of TS, SHOX-D, SGA, or ISS with impaired growth or short stature, according...

Countries:United StatesFranceGermanySouth KoreaSpain
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Recent Changes (Last 90 Days)
LOWMay 29, 2026NCT07221851lastUpdatePostDate: changed
LOWMay 29, 2026NCT07221851lastUpdatePostDate: changed
LOWMay 29, 2026NCT07221851lastUpdatePostDate: changed
LOWMay 26, 2026NCT07221851primaryCompletionDate: changed
LOWMay 26, 2026NCT05690386primaryCompletionDate: changed
LOWMay 24, 2026NCT07221851studyFirstPostDate: changed
LOWMay 24, 2026NCT05690386studyFirstPostDate: changed