Achondroplasia Treatments
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Brand Name
Yuviwel
Generic Name
Navepegritide
View Brand Information FDA approval date: February 27, 2026
Form: Kit
What is Yuviwel (Navepegritide)?
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. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial. YUVIWEL is a C-type natriuretic peptide analog 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. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial.
Approved To Treat
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Related Clinical Trials
A Phase 2b, Multicenter, Double-Blind, Randomized, Placebo-controlled Trial Evaluating Efficacy and Safety of Subcutaneous Doses of Navepegritide Administered Once Weekly for 52 Weeks in Adolescents (12-18 Years of Age) With Achondroplasia.
Summary: The purpose of this clinical trial is to evaluate efficacy and safety of once weekly subcutaneous (SC) doses of navepegritide 100 μg/kg compared to placebo (inactive drug) in adolescents aged 12 to 18 years with Achondroplasia. What will be measured is Annualized Growth Velocity after a 52-week treatment period.
A Phase 2, Multicenter, Double-Blind, Randomized, Placebo-controlled Trial, Evaluating Safety, Tolerability, and Efficacy of Subcutaneous Doses of TransCon CNP Administered Once Weekly for 52 Weeks in Infants (0 to <2 Years of Age) With Achondroplasia Followed by an Open Label Extension (OLE) Period
Summary: This trial is a Phase 2, multicenter, double-blind, randomized (ratio 2:1 TransCon CNP vs. placebo), placebo-controlled trial, designed to evaluate the safety, tolerability, and efficacy of 100 μg CNP/kg of Navepegritide (TransCon CNP) administered SC once-weekly for 52 weeks in infants with genetically verified heterozygous ACH, aged 0 to \< 2 years at the time of randomization.
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Brand Information
Yuviwel (Navepegritide)
1INDICATIONS AND USAGE
YUVIWEL
2DOSAGE FORMS AND STRENGTHS
For injection: 1.3 mg, 2.8 mg, and 5.5 mg of CNP(89-126) as a white to off-white lyophilized powder in a single-dose vial for reconstitution.
3CONTRAINDICATIONS
None.
4DESCRIPTION
YUVIWEL for injection contains navepegritide, a C-type natriuretic peptide (CNP) analog. Navepegritide is a prodrug of active CNP consisting of a CNP moiety transiently conjugated to two branched 20 kDa methoxy polyethylene glycol (mPEG) moieties via a proprietary TransCon
The molecular formula is C
YUVIWEL is provided as a sterile, lyophilized white to off-white powder for reconstitution to a colorless solution with Sterile Water for Injection, USP (diluent). The diluent for reconstitution of YUVIWEL is provided in a prefilled syringe.
The compositions of YUVIWEL are shown in Table 3.
5CLINICAL STUDIES
The effectiveness of YUVIWEL has been established in a clinical trial of navepegritide consisting of a randomized, double-blind, placebo-controlled 52-week period, followed by a single-arm 52-week OLE period (Trial 1; NCT05598320).
Trial 1 enrolled 84 treatment-naïve pediatric patients with genetically confirmed achondroplasia: 57 patients received navepegritide 0.1 mg/kg administered subcutaneously once weekly, and 27 received placebo.
The mean age at enrollment was 5.7 years (range: 2 to 12). Forty-five patients (54%) were male, and 39 patients (46%) were female. Overall, 74 patients (88%) were White, 8 patients (10%) were Asian, and 2 patients (2%) were mixed race. The patients had a mean baseline CDC-based height Z-score of -5.0 and mean baseline height of 89 cm (range: 64 to 120).
The primary efficacy endpoint was annualized growth velocity (AGV) at Week 52. Height Z-scores calculated using reference data from untreated children with achondroplasia (achondroplasia-specific height Z-score) and using reference data from the general population (CDC-based height Z-score) were also evaluated.
Treatment with once-weekly navepegritide for 52 weeks resulted in a least-squares mean treatment difference in AGV of 1.5 cm/year and a least-squares mean increase from baseline in achondroplasia-specific height Z-score of 0.3 (see
Improvements in AGV and height Z-scores were observed in navepegritide-treated patients across all predefined subgroups analyzed, including age group, sex, and region. In patients < 5 years of age, the least-squares mean treatment difference for AGV at Week 52 was 1.0 cm/year. In patients ≥ 5 years of age, the least-squares mean treatment difference was 1.8 cm/year.
Long-term Treatment Effect
All 57 patients in a 52-week randomized, double-blind, placebo-controlled Phase 2 dose-finding trial (Trial 2; NCT04085523) entered the single-arm OLE and continued treatment with navepegritide. AGV was maintained among those who had received 2 years of treatment with navepegritide 0.1 mg/kg once weekly.
6HOW SUPPLIED/STORAGE AND HANDLING
How Supplied
YUVIWEL (navepegritide) for injection is supplied as a lyophilized white to off-white powder in a vial, in three strengths: 1.3 mg, 2.8 mg, and 5.5 mg (see
Only use the vial, needles, and syringes that are in the Kits for handling of YUVIWEL
7PATIENT COUNSELING INFORMATION
Advise the patient and/or caregiver to read the FDA-approved patient labeling (Patient Information and Instructions for Use).
Administration Instructions
Provide appropriate instructions for injection to the patient/caregiver based on the YUVIWEL Instructions for Use (available at www.Yuviwel.com). Patients/caregivers and healthcare providers may also call the Ascendis Pharma Customer Support toll-free number at 1-844-442-7236 (1-844-44ASCENDIS) for assistance or additional training, if needed.
- Advise patients/caregivers to refer to the Instructions for Use that accompanies YUVIWEL for complete reconstitution and administration instructions with illustrations
- Advise patients/caregivers to administer YUVIWEL once weekly on the dosing day, at any time of day.
- Advise patients and caregivers to rotate the site of injection to reduce the risk of injection site reactions
Missed Dose [see
- Advise patients/caregivers that to avoid missed doses, YUVIWEL can be taken up to 2 days before or 2 days after the scheduled dosing day.
- If a dose is missed and more than 2 days have passed from the scheduled day, advise patients/caregivers to skip the missed dose and administer the next dose on the regularly scheduled day.
- To change the regular dosing day to a different day of the week, advise patients/caregivers to ensure that at least 5 days will elapse between the last dose and the newly established regular dosing day.
Risk of Low Blood Pressure
Advise patients and/or caregivers to contact their healthcare provider if they experience symptoms of decreased blood pressure (e.g., dizziness, fatigue, and/or nausea) while being treated with YUVIWEL
8INSTRUCTIONS FOR USE
Yuviwel
Ascendis Pharma
For Use Under the Skin Only (subcutaneous)
1 time a week injection
This leaflet contains:
Additional information
If you need help, please ask your healthcare provider for advice. You may also call the Ascendis Pharma Customer Support toll-free number at 1-844-442-7236 (1-844-44ASCENDIS) for assistance or additional training, if needed.
Yuviwel.com
This Instructions for Use contains information on how to prepare and inject Yuviwel.
Parts Overview
Step 1
Important:
Step 2 and Step 3
Important:
Supplies Not Included:
You will also need a sharps disposal container and alcohol wipes.
Get to know Yuviwel
Make sure you have received training from your healthcare provider before injecting.
Read and follow the Instructions for Use step-by-step to make sure you inject Yuviwel the right way.
Important: Prior to use, read the Instructions for Use carefully even if you have used a similar product. The steps may not be the same.
If you are blind or visually impaired, do not use Yuviwel without help from a person who is trained to use it.
How Yuviwel is supplied and injected
Yuviwel is injected under the skin (subcutaneous injection) 1 time a week.
Each Yuviwel carton includes 4 Kits, with 1 Prescribing Information and 1 Instructions for Use.
Yuviwel is available in the following strengths:
Always check that you inject the dose strength prescribed by your healthcare provider.
If you are injecting yourself with Yuviwel, you should use the stomach area (2 inches from belly button) or thighs as injection sites.
If someone else gives you the Yuviwel injection, the buttocks or the back of the upper arms can be used as injection sites.
Only inject into injection sites instructed by your healthcare provider.
Important before starting Yuviwel
Check all parts and their packaging for signs of damage.
Do not use if any parts or packaging appear damaged. Use a new Kit.
Your healthcare provider may prescribe a dose that is more than 1 milliliter (mL) and requires the use of two Kits to deliver two injections for a complete dose.
If you have been prescribed a dose that is more than 1 mL:
- Prepare and give your first injection, using one Kit (follow Steps 1 through 3.10)
- Then, using a new Kit, immediately prepare and give a second injection by repeating steps 1 through 3.10.
Check the expiration date on the Kit.
Only use the vial, needles, and syringes that come in your Kit.
If you do not have an alcohol wipe, use soap and water to clean the injection site and the grey rubber at the top of the vial.
The vial, needles, and syringes in the Kit are for
Do not reuse any of the parts. Do not recap needles.
Be careful when handling needles to reduce the risk of needlestick injury and infection.
Do not throw away opened or used needles in your household trash. Use a sharps disposal container.
After unpacking and to
Do not change the injection angle after the needle has been inserted into the skin. Changing the angle can cause the needle to bend or break off under the skin. If a needle breaks in your skin, get medical help right away.
Throwing away Yuviwel parts
Throw away (dispose of) used needles, syringes and the vial in an FDA-cleared sharps disposal container right away after use.
- Do not throw away (dispose of) used needles and syringes in the household trash.
- Do not reuse needles. Always use a new needle for each injection.
If you do not have an FDA-cleared sharps disposal container, you may use a household container that is:
- Made of heavy-duty plastic,
- Can be closed with a tight-fitting, puncture resistant lid, without sharps being able to come out,
- Upright and stable during use,
- Leak-resistant, and
- Properly labeled to warn of hazardous waste inside the container
When your sharps disposal container is almost full, you will need to follow your community guidelines for the right way to dispose of your sharps disposal container. There may be state or local laws about how you should throw away used needles and syringes.
For more information about safe sharps disposal, and for specific information about sharps disposal in the state that you live in, go to the FDA's website at: www.fda.gov/safesharpsdisposal
Storing Yuviwel
Store Yuviwel refrigerated between 36°F to 46°F (2°C to 8°C) in the original carton to protect from light. Do not freeze. Yuviwel can also be stored at room temperature up to 86°F (30°C) for up to 6 months and can be returned to the refrigerator within the 6 months.
Do not use Yuviwel past the expiration date or 6 months after the date it was first removed from the refrigerator (whichever comes first).
Keep Yuviwel and all medicines out of the reach of children. Small parts in the Kit may cause a choking hazard for children and pets.
Yuviwel does not contain preservatives.
Step 1
Gather supplies:
1 Kit, 2 alcohol wipes and a sharps disposal container.
Note: If the Kit has been stored in the refrigerator, let the vial and Prefilled Diluent Syringe sit at room temperature for about 30 minutes before mixing (reconstitution).
Step 2
Prepare injection
Step 3
Important
If your Yuviwel
If your prescribed dose is more than 1 mL, get a new Kit and repeat steps 1 through 3.10 to give a second injection for a complete dose. The second injection must be given right after the first injection,
Remember to choose a different injection site for each Yuviwel injection.
Do not reuse any parts for the 2 injections.
This Instructions for Use has been approved by the U.S. Food and Drug Administration
9PRINCIPAL DISPLAY PANEL - 1.3 mg Kit Carton
NDC 73362-201-01
yuviwel
1.3 mg/vial
For Subcutaneous Use Only
Prior to Use, read the Instructions for Use
This carton contains 4 Kits and 1 Patient Information.
- 1 single-dose vial of Yuviwel 1.3 mg/vial
- 1 prefilled diluent syringe containing 0.8 mL Sterile Water
- 2 single use preparation needles
- 1 single use injection needle
- 1 single use injection syringe
- 1 Instructions for Use
- 1 Prescribing Information
10PRINCIPAL DISPLAY PANEL - 2.8 mg Kit Carton
NDC 73362-202-01
yuviwel
2.8 mg/vial
For Subcutaneous Use Only
Prior to Use, read the Instructions for Use
This carton contains 4 Kits and 1 Patient Information.
- 1 single-dose vial of Yuviwel 2.8 mg/vial
- 1 prefilled diluent syringe containing 0.8 mL Sterile Water
- 2 single use preparation needles
- 1 single use injection needle
- 1 single use injection syringe
- 1 Instructions for Use
- 1 Prescribing Information
11PRINCIPAL DISPLAY PANEL - 5.5 mg Kit Carton
NDC 73362-203-01
yuviwel
5.5 mg/vial
For Subcutaneous Use Only
Prior to Use, read the Instructions for Use
This carton contains 4 Kits and 1 Patient Information.
- 1 single-dose vial of Yuviwel 5.5 mg/vial
- 1 prefilled diluent syringe containing 1.1 mL Sterile Water
- 2 single use preparation needles
- 1 single use injection needle
- 1 single use injection syringe
- 1 Instructions for Use
- 1 Prescribing Information
