Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Navepegritide · 3 trials · 1 indication
cm per year
Incidence of treatment emergent adverse events (TEAEs)
Change from baseline to 52 weeks in length/height Z-score
Annualized growth velocity is defined as (height - baseline height)/(date of height assessment - date of baseline) \* 365.25. Annualized growth velocity reported in terms of centimeters (cm) per year. Missing values at Week 52 were imputed by a multiple imputation method.
| Arm | Type | Description |
|---|---|---|
| Navepegritide | EXPERIMENTAL | Once weekly double-blinded treatment with SC injection of 100 µg/kg of navepegritide for 52 weeks |
| Placebo for Navepegritide | PLACEBO_COMPARATOR | Once weekly double-blinded treatment with SC injection of 100 µg/kg of placebo for navepegritide for 52 weeks |
| Open-Label Extension Period: Navepegritide | EXPERIMENTAL | Participants who completed the 52-week treatment period continued into the open-label extension period and received treatment with navepegritide (TransCon CNP) doses up to Week 104. All participants received navepegritide at a dose of 100 μg/kg/week. |
| Name | Type | Description |
|---|---|---|
| Navepegritide | DRUG | Once-weekly subcutaneous injection of 100 µg/kg navepegritide for 52 weeks |
| Placebo for navepegritide | DRUG | Once-weekly subcutaneous injection of 100 µg/kg placebo for navepegritide |
| Navepegritide (TransCon CNP) | DRUG | Once-weekly subcutaneous injection of 100 µg/kg Navepegritide (TransCon CNP) |
| Placebo for Navepegritide (TransCon CNP) | DRUG | Once-weekly subcutaneous injection of 100 µg/kg placebo for Navepegritide (TransCon CNP) |
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...
Navepegritide is an investigational drug being developed for achondroplasia, a rare genetic condition that causes short stature and disproportionate dwarfism. It is currently in Phase 2 clinical trials evaluating its efficacy and safety in children, infants, and adolescents with achondroplasia.
Navepegritide is a small molecule designed to treat achondroplasia. Its specific molecular target is not disclosed in the available information. The drug is being studied for its ability to improve growth outcomes in patients with achondroplasia.
Navepegritide is being developed by Ascendis Pharma A/S, a biopharmaceutical company listed on the stock exchange under the ticker ASND. The company is conducting Phase 2 clinical trials to evaluate the drug's safety and efficacy in treating achondroplasia.
Navepegritide is currently in Phase 2 clinical development. It is an investigational drug, meaning it has not been approved by regulatory authorities. The ongoing trials are designed to assess its efficacy and safety in different age groups with achondroplasia.
Navepegritide is being studied in three Phase 2 clinical trials. NCT05598320, completed, evaluated TransCon CNP in children aged 2 years and older. NCT06079398, recruiting, is for infants under 2 years. NCT06732895, recruiting, is for adolescents aged 12-18 years.
Navepegritide is also known as TransCon CNP, as indicated by the clinical trial titles. It is administered as two separate subcutaneous injections in combination with lonapegsomatropin. The drug is being evaluated for treating achondroplasia across different age groups.