Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Label indication YUVIWEL ® is indicated to increase linear growth in pediatric patients 2 years of age and older with achondroplasia with open epiphyses. This indication is approved under accelerated approval based on an improvement in annualized growth velocity [see Clinical Studies (14) ]. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s). FDA label
Also known as Navepegritide (TransCon CNP)
Navepegritide · 4 trials · 1 indication
cm per year
safety profile of navepegritide and lonapegsomatropin
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 |
| Combination of Navepegritide and Lonapegsomatropin | EXPERIMENTAL | - |
| 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 |
| Combination of Navepegritide and Lonapegsomatropin administered as two separate s.c. injections | DRUG | For navepegritide, a once weekly s.c. dose of 100 μg CNP/kg. For lonapegsomatropin, a once weekly s.c. dose of lonapegsomatropin 0.30 mg hGH/kg as starting dose. Treatment duration of up to 156 weeks. |
| 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 therapy being developed for achondroplasia, the most common form of disproportionate short stature. It is also known as TransCon CNP. Ascendis Pharma A/S is developing the drug, and it is currently in Phase 2 clinical development.
Navepegritide is being studied for the treatment of achondroplasia in people with the condition. Clinical trials have enrolled infants, children, and adolescents with achondroplasia. It is investigational and has not been approved for any use.
Navepegritide is developed by Ascendis Pharma A/S, which trades under the ticker ASND. The company is running a Phase 2 program for the drug in achondroplasia, including studies in infants, children, and adolescents.
Navepegritide is in Phase 2 clinical development for achondroplasia. It is investigational and has not been approved by the FDA. Four trials have been conducted or are ongoing, with three currently active and one completed.
Navepegritide trials include NCT06732895 in adolescents aged 12 to 18, NCT06433557 testing it in combination with lonapegsomatropin in children, NCT06079398 in infants under 2 years, and the completed NCT05598320 in children. Total enrollment across these studies is 203 participants.
Yes, navepegritide is also known as TransCon CNP. Some trials also study navepegritide in combination with lonapegsomatropin, given as two separate subcutaneous injections. Both names refer to the same Ascendis Pharma development program for achondroplasia.