Navepegritide
InvestigationalLong-acting CNP prodrug
Also known as: TransCon CNP
A real evidence base, with gaps
- Regulatory standing. In clinical trials, not yet approved
- Limited human data. Some human data exists, not conclusive
- Registered human trials. 5 registered studies on ClinicalTrials.gov, most advanced Phase 3
- No sources. Nothing cited to verify
Computed with the same four public criteria as every profile. The human-evidence grade on an indexed profile is set automatically and may change when it is hand-reviewed. It is not a safety score, not a purity rating, and not medical advice, and not a claim that this compound treats, cures, or prevents any condition.
What it is
Navepegritide, also called TransCon CNP, is an investigational long-acting prodrug of C-type natriuretic peptide (CNP) developed by Ascendis Pharma. It uses a carrier system designed to release active CNP slowly, which is thought to counteract overactive FGFR3 signaling in growth plates and support bone growth in achondroplasia.
What the evidence shows
It is not FDA-approved; about five registered human trials exist, reaching Phase 3 in children with achondroplasia, with published results suggesting increased growth velocity. Long-term effects on final height and other outcomes remain unestablished.
Registered human trials
Live from ClinicalTrials.gov, refreshed daily.
A Phase 2 Clinical Trial to Evaluate Efficacy, Safety, and Tolerability of Navepegritide in Combination With Lonapegsomatropin in Children With Achondroplasia
A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Children With Achondroplasia
A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Infants (0 to <2 Years of Age) With Achondroplasia
A Clinical Trial to Evaluate Efficacy and Safety of Navepegritide in Adolescents (12 - 18 Years of Age) With Achondroplasia.
Published literature
Live from Europe PMC. This is how much has been published, not how much is proven in humans.
Once-weekly TransCon CNP (navepegritide) in children with achondroplasia (ACcomplisH): a phase 2, multicentre, randomised, double-blind, placebo-controlled, dose-escalation trial.
Once-Weekly Navepegritide in Children With Achondroplasia: The APPROACH Randomized Clinical Trial.
Achondroplasia treatments in children aged 5 and older.
Progress in managing children with achondroplasia.
Sources
Risks, side effects and study doses are only published on hand-reviewed profiles. Want this one reviewed, or spotted something wrong? Tell us.