Voxzogo
Voxzogo (vosoritide) is the first medication approved to treat achondroplasia, a genetic condition that leads to slowed bone growth. This medication should only be given to children age 5 years and older whose growth plates are still open. Parents and caregivers might find it difficult to give this medication to their child because it needs to be injected under the skin daily. Voxzogo (vosoritide) was approved by the FDA through its Accelerated Approval Program. It's only available at specialty pharmacies, so your child's provider or pharmacist will need to help you set that up.
What is Voxzogo (vosoritide)?
What is Voxzogo (vosoritide) used for?
- Growth in children 5 years and older who still have potential to grow (achondroplasia with open growth plates, or epiphyses)
How Voxzogo (vosoritide) works
Voxzogo (vosoritide) is a man-made version of a protein called C-type natriuretic peptide (CNP).
In your body, there are 2 proteins that work together at your growth plate to help your bones grow properly. CNP signals your bones to grow. Another protein called fibroblast growth factor receptor 3 (FGFR3) slows bone growth. Both CNP and FGFR3 need to work in balance to help you grow.
If you have achondroplasia, you have too much FGFR3 and not enough CNP. As a result, your growth is stunted. Voxzogo (vosoritide) works alongside natural CNP in your body to improve the balance with FGFR3, which helps promote bone growth.
Drug facts
Side effects of Voxzogo (vosoritide)
The following side effects may get better over time as your body gets used to the medication. Let your healthcare provider know immediately if you continue to experience these symptoms or if they worsen over time.
Common Side Effects
- Redness or warmth at injection site (75%)
- Swelling at injection site (62%)
- Vomiting (27%)
- Hives at injection site (25%)
- Joint pain (15%)
- Dizziness, fainting or other symptoms of low blood pressure (13%)
- Stomach pain, nausea, or other symptoms of stomach flu (13%)
Less Common Side Effects
- Diarrhea
- Dizziness
- Ear pain
- Fever, body aches, or other symptoms of the flu
- Feeling tired
- Runny nose, watery eyes, or other symptoms of seasonal allergy
- Dry skin
The following Voxzogo (vosoritide) side effects have also been reported
Side effects that you should report to your care team as soon as possible:
- Allergic reactions—skin rash, itching, hives, swelling of the face, lips, tongue, or throat
- Low blood pressure—dizziness, feeling faint or lightheaded, blurry vision
Side effects that usually do not require medical attention (report these to your care team if they continue or are bothersome):
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Pros and cons of Voxzogo (vosoritide)
Pros
- The first and only FDA-approved medication for achondroplasia
- Shown to improve growth rates in children with achondroplasia for 2 years
- Give once daily
Cons
- Only available as a brand medication, so it might be expensive
- Need to mix and draw up the medication before using, so it might be difficult to use for certain people
- Not recommended for use in people with severe kidney disease
Pharmacist tips for Voxzogo (vosoritide)
- Read the instructions for use or watch the instructional video on how to prepare and give Voxzogo (vosoritide) injections. Don't give your child Voxzogo (vosoritide) injections until you understand how to prepare and inject a dose. Your child's provider will show you how to give your child's first injection. If you have questions, contact your provider.
- Store Voxzogo (vosoritide) in the refrigerator before mixing. Keep the medication in its original container to protect it from light. Don't use this medication if it's expired.
- You can store unmixed Voxzogo (vosoritide) at room temperature, but it's only good for 90 days after you move it from the refrigerator to room temperature. Mark the date you moved the medication to room temperature so you can keep track of the 90 days.
- When you're ready to prepare and give Voxzogo (vosoritide), wash your hands with soap and water to prevent contamination (infection).
- After mixing Voxzogo (vosoritide) with sterile water, keep the mixture at room temperature. Make sure to use the medication within 3 hours. Don't use the mixture if it's cloudy or has particles.
- Only use sterile water, syringes, or needles provided by your pharmacy. Don't share or reuse needles or syringes. Doing so can put your child at risk for getting or transmitting infections. Throw away the vials, needles, and syringes in a sharps disposal container after each use.
- Make sure your child has enough to eat and drink (8-10 ounces of fluid) within an hour before receiving Voxzogo (vosoritide). Doing so can help prevent this medication from lowering your child's blood pressure too much and causing dizziness or fainting. Consider giving Voxzogo (vosoritide) right after your child's meal time.
- If your child feels dizzy or faint after their Voxzogo (vosoritide) injection, let their provider know. Also ask your child to lay down on their back with their legs raised to ease these symptoms.
- Tell your provider if your child experiences any changes in weight while receiving Voxzogo (vosoritide) because the dose might need to be changed. Let your provider know if your child has nausea, vomiting, or diarrhea that won’t go away since this can also be a cause of weight loss.
- Make sure your child gets routine physical checkups done on time while they're receiving Voxzogo (vosoritide). Your child's provider will want to monitor your child's growth every 3-6 months while using this medication to make sure it's working.
Common questions about Voxzogo (vosoritide)
The FDA's Accelerated Approval Program allows a new medication to be approved faster than usual if it fulfills an unmet need or if it treats serious or life-threatening conditions. These approvals are based on lab measurements that are thought to predict health outcomes before the medication shows clear benefits. For Voxzogo (vosoritide), the medication helps children with achondroplasia grow taller faster, which can potentially predict a better rate of height growth in children with achondroplasia. After accelerated approval, the manufacturer is required to complete clinical studies to confirm beneficial health outcomes (in this case, if Voxzogo (vosoritide) improved final height in people with achondroplasia). Depending on the results of these studies, the FDA can either give the medication a full approval or pull it off the market.
Voxzogo (vosoritide) is used in children 5 years and older living with achondroplasia. It hasn't been studied in children younger than 5 years of age. This medication only works well in children whose growth plates are still open, meaning they still have potential to grow. Growth plates typically close for most children sometime between the puberty years and the end of the teenage years. Your child's provider will monitor your child's growth and development every 3-6 months to make sure this medication is working for them. Once the growth plates are closed, your provider will stop treatment with Voxzogo (vosoritide) since it might not be helpful anymore. If you have any questions about whether Voxzogo (vosoritide) is right for your child, follow up with your child's provider for more information.
Voxzogo (vosoritide) can be injected under the skin on the belly, buttocks, upper arms, and thighs. Make sure to avoid the area 2 inches within the belly button. Rotate a different injection site every day to prevent skin irritation. Don't inject the medication into skin that's swollen, red, or tender.
If your child missed a dose of Voxzogo (vosoritide) and it’s still within 12 hours of the usual time they receive the injection, you can give them their dose as soon as possible. But if more tha 12 hours has past since their usual injection time, then skip the missed dose. Give the next dose of Voxzogo (vosoritide) on your child's next scheduled day and time.
Typically, Voxzogo (vosoritide) should be stored in the refrigerator before opening. If you leave it out of the refrigerator, you can still use it up to 90 days from the date it was left out, as long as it hasn't been mixed. Make sure you write down the date the vial was first at room temperature on the label to keep track. Don't put Voxzogo (vosoritide) back in the refrigerator after it's been left out. Throw away any unused medication after 90 days of storing it at room temperature.
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Risks and warnings for Voxzogo (vosoritide)
Voxzogo (vosoritide) can cause some serious health issues. This risk may be even higher for certain groups. If this worries you, talk to your doctor or pharmacist about other options.
Low blood pressure
- Risk factors: People who take blood pressure medications | People with heart problems
Your child might experience a temporarily drop in your blood pressure after the Voxzogo (vosoritide) injection. Their risk is higher if they're taking blood pressure medications or have a history of heart problems. To lower the risk of low blood pressure, make sure your child stays hydrated and has eaten before receiving Voxzogo (vosoritide) injection. If your child experiences dizziness, low energy, or nausea after the injection, tell your child's provider right away.
Voxzogo (vosoritide) dosage
Kit| Dosage | Quantity | Price as low as | Price per unit |
|---|---|---|---|
| 0.4mg of | 3 kits | $32,754.73 | $10,918.24 |
| 0.56mg | 3 kits | $32,754.73 | $10,918.24 |
| 1.2mg of | 3 kits | $32,754.73 | $10,918.24 |
Typical dosage for Voxzogo (vosoritide)
Your child's provider will determine the dose of Voxzogo (vosoritide) your child needs based on their body weight.
Children 5 years and older: The typical dose of Voxzogo (vosoritide) ranges from 0.24 mg to 0.8 mg based injected under the skin once daily.
