Brand Name
Voxzogo
Generic Name
Vosoritide
View Brand Information FDA approval date: November 19, 2021
Form: Kit
What is Voxzogo (Vosoritide)?
VOXZOGO is indicated to increase linear growth in pediatric patients 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. VOXZOGO is a C type natriuretic peptide analog indicated to increase linear growth in pediatric patients 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 2, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Safety and Efficacy of Vosoritide in Infants and Young Children With Hypochondroplasia, Aged 0 to < 36 Months
Summary: The purpose of this study is to evaluate the safety and efficacy of daily administration of vosoritide in participants with HCH aged 0 to \< 36 months over a 52-week period.
A Phase 2, Randomized, Human Growth Hormone-Controlled, Multicenter, Basket Study of Vosoritide in Children With Turner Syndrome, Short Stature Homeobox-Containing Gene Deficiency, and Noonan Syndrome With an Inadequate Response to Human Growth Hormone. (CANOPY NS, TS, SHOX-D-2)
Summary: The purpose of this basket study in children with Turner syndrome, SHOX deficiency, and Noonan syndrome is to evaluate the effect of 3 doses of vosoritide versus hGH on growth as measured by AGV after 6 months of treatment. The long-term efficacy and safety of vosoritide at the therapeutic dose will be evaluated up to FAH.
VIrtual STudy in Achondroplasia for the US (VISTA)
Summary: This is an observational study of individuals with achondroplasia in the United States. The primary study population consists of pediatric individuals treated and untreated with VOXZOGO™. Study enrollment started in February 2023. The projected total duration of the study is approximately 5 years at minimum from start of study recruitment in February 2023, with the duration of individual prospecti...
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Brand Information
VOXZOGO 0.4mg (vosoritide)
1INDICATIONS AND USAGE
VOXZOGO is indicated to increase linear growth in pediatric patients with achondroplasia with open epiphyses. This indication is approved under accelerated approval based on an improvement in annualized growth velocity
2DOSAGE FORMS AND STRENGTHS
For Injection: 0.4 mg, 0.56 mg, or 1.2 mg of vosoritide as a white to yellow lyophilized powder in a single-dose vial for reconstitution.
3CONTRAINDICATIONS
None
4ADVERSE REACTIONS
The following clinically significant adverse reactions are described elsewhere in the labeling:
- Risk of Low Blood Pressure
4.1Clinical Trials Experience
Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice.
4.2Postmarketing Experience
The following adverse reactions have been identified during post approval use of VOXZOGO. Because these reactions are reported voluntarily from a population of uncertain size, it is not always possible to reliably estimate their frequency or establish a causal relationship to drug exposure.
Skin and Subcutaneous Tissue Disorders: hypertrichosis (includes the preferred terms: hair growth abnormal and hypertrichosis).
5DESCRIPTION
VOXZOGO contains vosoritide, a human C type natriuretic peptide (CNP) analog. Vosoritide is a 39 amino acid peptide. Its amino acid sequence includes the 37 C terminal amino acids of the human CNP53 sequence plus Pro Gly on the N terminus to convey resistance to neutral endopeptidase (NEP) degradation. Vosoritide is manufactured from
Vosoritide has the structural formula shown in Figure 1.
Figure 1
VOXZOGO (vosoritide) for injection, is a sterile, preservative-free white-to-yellow lyophilized powder, for subcutaneous administration after reconstitution with Sterile Water for Injection, USP.
VOXZOGO is provided as a single-dose vial containing 0.4 mg, 0.56 mg, or 1.2 mg of vosoritide per vial. A pre-filled syringe containing Sterile Water for Injection, USP for use as a diluent is also provided. The contents of each single dose vial are summarized by strength in Table 4. The product contains no preservative.
6PATIENT COUNSELING INFORMATION
Advise the patient and caregiver to read the FDA-approved patient labeling (Patient Information and Instructions for Use).
7INSTRUCTIONS FOR USE VOXZOGO™ [Vox zoeʹ goe] (vosoritide) for injection, for subcutaneous use Single-Use
This Instructions for Use contains information for caregivers on how to inject VOXZOGO.
Read this Instructions for Use before you start using VOXZOGO and each time you get a refill. There may be new information. This information does not take the place of talking to your child's healthcare provider about your child's medical condition and their treatment. Before you use VOXZOGO for the first time, make sure your child's healthcare provider shows you the right way to use it. Contact your child's healthcare provider if you or your child have any questions.
Important Information You Need to Know Before Injecting VOXZOGO
- Wash your hands with soap and water.
- Do not drop VOXZOGO or put opened items down on surfaces that are not clean.
- VOXZOGO is available in more than 1 strength.
- Take the VOXZOGO vial and prefilled diluent syringe out of the refrigerator and allow them to reach room temperature before mixing.
- Inspect the vial and supplies for any signs of damage or contamination. Do not use if damaged or contaminated.
- Check the expiration date. The expiration date can be found on the carton, vial and prefilled diluent syringe. Do not use if expired.
- Your child should eat a meal and drink a glass (about 8 to 10 ounces) of fluid (such as water, milk, or juice) within 1 hour before injection.
- VOXZOGO should be given at about the same time every day.
- Do not mix VOXZOGO with other medicines.
- After mixing the VOXZOGO, use it right away. Do not use the mixed VOXZOGO if it has been sitting at room temperature for more than 3 hours. Throw it away (dispose of) in a sharps container. See step 18 and " for more information.
- Do not reuse any of the supplies. After the injection, throw away (dispose of) the used vial even if there is VOXZOGO remaining. See step 18 and " for more information.
How to Store VOXZOGO
- Store the VOXZOGO vial and prefilled diluent syringe in the refrigerator between 36°F to 46°F (2°C to 8°C).
- You may store VOXZOGO (before mixing) at room temperature between 68°F to 77°F (20°C to 25°C) for 90 days. Record the date you started storing VOXZOGO at room temperature on the carton to keep track of the 90 days.
- Do not freeze VOXZOGO.
- Store VOXZOGO out of direct sunlight.
Keep VOXZOGO and all other medicines out of the reach of children.
Supplies Needed to Inject VOXZOGO
Gather all of these supplies on a clean, flat surface before injecting.
Preparing VOXZOGO for Injection
Select and Prepare Injection Site
Giving VOXZOGO Injection
How to Throw Away (Dispose of) VOXZOGO
Put your used or expired vials, needles and syringes in a FDA-cleared sharps disposal container right away after use.
If you do not have a FDA-cleared sharps disposal container, you may use a household container that:
- is made of a heavy-duty plastic,
- can be closed with a tight-fitting, puncture-resistant lid without sharps being able to come out,
- is upright and stable during use,
- is leak-resistant, and
- is 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 local or state 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:
Do not dispose of your used sharps disposal container in your household trash unless your community guidelines permit this. Do not recycle your used sharps disposal container.
After the Injection
- Inspect the injection site. If a small amount of bleeding occurs from the injection site, gently press a gauze pad on it for a few seconds or apply a bandage.
- Monitor for signs of low blood pressure, such as dizziness, tiredness, and nausea. If your child experiences these symptoms you should call your child's healthcare provider, then get your child to lay back with legs raised.
For Help or More Information
- Call your healthcare provider
- Call BioMarin at 1-800-123-4567
- Visit www.VOXZOGO.com
Manufactured for:
REP-5233-C10
This Instructions for Use has been approved by the U.S. Food and Drug Administration.
8PRINCIPAL DISPLAY PANEL - Kit Carton - 68135-082
NDC 68135-082-36
Rx Only
VOXZOGO™
0.4 mg per vial
For Subcutaneous Use Only
This carton contains:
Must be reconstituted
BiOMARIN
9PRINCIPAL DISPLAY PANEL - 0.4 mg Vial Label
VOXZOGO™
0.4 mg per vial
Reconstitute Prior to Use
For Subcutaneous Use Only
Single-dose vial
LOT:
EXP:
10PRINCIPAL DISPLAY PANEL - 0.5 mL Syringe Label
NDC 68135-070-12
Diluent
0.5 mL
For drug diluent use only-
Single-dose
No preservative
Distributed by: BioMarin
MLC7246001
LOT:
EXP:
10008821-02
11PRINCIPAL DISPLAY PANEL - Kit Carton - 68135-119
NDC 68135-119-66
Rx Only
VOXZOGO™
0.56 mg per vial
For Subcutaneous Use Only
This carton contains:
Must be reconstituted
BiOMARIN
12PRINCIPAL DISPLAY PANEL - 0.56 mg Vial Label
VOXZOGO™
0.56 mg per vial
Reconstitute Prior to Use
For Subcutaneous Use Only
Single-dose vial
LOT:
EXP:
13PRINCIPAL DISPLAY PANEL - 0.7 mL Syringe Label
NDC 68135-102-43
Diluent
0.7 mL
For drug diluent use only-
Single-dose
No preservative
Distributed by: BioMarin
MLC7246001
LOT:
EXP:
10008843-02
14PRINCIPAL DISPLAY PANEL - Kit Carton - 68135-181
NDC 68135-181-93
Rx Only
VOXZOGO™
1.2 mg per vial
For Subcutaneous Use Only
This carton contains:
Must be reconstituted
BiOMARIN
15PRINCIPAL DISPLAY PANEL - 1.2 mg Vial Label
VOXZOGO™
1.2 mg per vial
Reconstitute Prior to Use
For Subcutaneous Use Only
Single-dose vial
LOT:
EXP:
16PRINCIPAL DISPLAY PANEL - 0.6 mL Syringe Label
NDC 68135-158-17
Diluent
0.6 mL
For drug diluent use only-
Single-dose
No preservative
Distributed by: BioMarin
MLC7246201
LOT:
EXP:
10008822-02
