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Navepegritide

Phase 2

Achondroplasia | Small molecule | Other |Ascendis Pharma A/S|Last Updated: Apr 21, 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
RandomizedDouble-BlindPLACEBO_CONTROLLEDDMCBiomarker
Total Trials2
Total Enrollment96
FDA Designations
No designations recorded
Clinical Trials (2)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06732895A Clinical Trial to Evaluate Efficacy and Safety of Navepegritide in Adolescents (12 - 18 Years of Age) With Achondroplasia.PHASE2 RECRUITING 24Dec 10, 2024Sep 1, 2027Apr 21, 20265 Canada, Denmark +3
NCT06079398A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Infants (0 to <2 Years of Age) With AchondroplasiaPHASE2 RECRUITING 72Jan 23, 2024Dec 1, 2028Apr 8, 202618 United States, Australia +13
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Study Endpoints
Primary Endpoints
Annualized Growth Velocity
52 weeks

cm per year

To evaluate the safety and tolerability of Navepegritide
52 weeks

Incidence of treatment emergent adverse events (TEAEs)

To evaluate the effect of Navepegritide on growth
52 weeks

Change from baseline to 52 weeks in length/height Z-score

Secondary Endpoints
Height Z-score
52 weeks
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
NavepegritideEXPERIMENTALOnce weekly double-blinded treatment with SC injection of 100 µg/kg of navepegritide for 52 weeks
Placebo for NavepegritidePLACEBO_COMPARATOROnce weekly double-blinded treatment with SC injection of 100 µg/kg of placebo for navepegritide for 52 weeks
Interventions
NameTypeDescription
NavepegritideDRUGOnce-weekly subcutaneous injection of 100 µg/kg navepegritide for 52 weeks
Placebo for navepegritideDRUGOnce-weekly subcutaneous injection of 100 µg/kg placebo for navepegritide
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Eligibility Criteria
Age Range12 Years — 17 Years
SexALL
Healthy VolunteersNo
Study Sites5

Inclusion Criteria: * Written, signed informed consent and/or assent of the participant, participant parent(s) or legal guardian(s) of the participant, and as required by the institutional review board/human research ethics committee/independent ethics committee (IRB/HREC/IEC). For participants who...

Countries:CanadaDenmarkFranceIrelandUnited KingdomUnited StatesAustraliaAustriaFinlandGermanyItalyNew ZealandNorwayPortugalSweden
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT06079398primaryCompletionDate: changed
MEDIUMMay 26, 2026NCT06732895primaryCompletionDate: changed
LOWMay 24, 2026NCT06079398studyFirstPostDate: changed
LOWMay 24, 2026NCT06732895studyFirstPostDate: changed