Vosoritide

WHY is this medicine prescribed?

Vosoritide is used to increase linear growth (height) in certain children who have achondroplasia (ACH; achondroplastic dwarfism; a genetic condition of bone growth that results in short arms and legs). Vosoritide is in a class of medications called C type natriuretic peptide (CNP) analogs. It works by increasing cartilage cell growth which results in increased bone growth.

Are there OTHER USES for this medicine?

This medication may be prescribed for other uses; ask your doctor or pharmacist for more information.

HOW should this medicine be used?

Vosoritide comes as a powder to be mixed with liquid to inject subcutaneously (just under the skin). It is usually injected once a day. Inject vosoritide injection at around the same time every day. Follow the directions on your prescription label carefully, and ask your doctor or pharmacist to explain any part you do not understand. Use vosoritide injection exactly as directed. Do not use more or less of it or take it more often than prescribed by your doctor.

Vosoritide injection may cause a temporary decrease in blood pressure. Your child should eat a meal and drink 8 to 10 ounces of liquid such as water, milk, or juice within 60 minutes before receiving vosoritide injection. Call your doctor or nurse if your child experiences any of the following symptoms that may occur after the injection: dizziness, tiredness, or nausea.

Your healthcare provider will monitor your child's growth carefully during treatment with vosoritide injection and will adjust the dose and the length of treatment based on your child's response to this medication.

If you will be injecting vosoritide injection by yourself at home or having a friend or relative inject the medication for you, your doctor will show you or the person who will be injecting the medication how to mix the medication properly and how to inject it. Before you use vosoritide injection yourself the first time, carefully read the written instructions for use that come with the medication. Ask your doctor or pharmacist if you have any questions about how to mix the medication or do not understand how to inject vosoritide.

Before you are ready to inject vosoritide, you will need to remove the vial of vosoritide and the prefilled diluent syringe from the refrigerator and allow them to warm to room temperature.

You should always look at vosoritide injection solution (liquid) after mixing and before you inject it. The solution should be clear and colorless to yellow, with no particles in it. Do not use vosoritide injection solution if it is colored, cloudy, contains particles, or if the expiration date on the vial has passed.

Use each syringe only once. Dispose of used syringe in a puncture-resistant container. Talk to your doctor or pharmacist about how to dispose of the puncture-resistant container.

Inject vosoritide into the front of the thighs, buttocks, or anywhere on your stomach except your navel (belly button) and the area 2 inches around it. If another person is injecting your medication, the outer area of the upper arms also may be used. Do not inject the medication into skin that is tender, bruised, damaged, or scarred. Choose a different spot each time you inject the medication.

Ask your pharmacist or doctor for a copy of the manufacturer's information for the patient.

What SPECIAL PRECAUTIONS should I follow?

Before receiving vosoritide injection,

• tell your doctor and pharmacist if you are allergic to vosoritide, any other medications, or any of the ingredients in vosoritide injection. Ask your pharmacist for a list of the ingredients.

• tell your doctor and pharmacist what other prescription and nonprescription medications, vitamins, nutritional supplements you are taking or plan to take. Be sure to mention any of the following: blood pressure medications.

• tell your doctor if you have or have ever had heart or kidney problems.

• tell your doctor if you are pregnant or are breast-feeding. If you become pregnant while receiving vosoritide injection, call your doctor.

What SPECIAL DIETARY instructions should I follow?

Unless your doctor tells you otherwise, continue your normal diet.

What should I do IF I FORGET to take a dose?

If it is 12 hours or less after you miss a dose of vosoritide, inject the dose as soon as you remember it. However, if it is more than 12 hours after your missed dose, skip the missed dose and continue your regular dosing schedule. Do not use a double dose to make up for a missed one.

What SIDE EFFECTS can this medicine cause?

Vosoritide may cause side effects. Tell your doctor if any of these symptoms are severe or do not go away:

• redness, rash, hives, itching, swelling, pain, or bruising near or at the spot that the medication was injected
• nausea
• dizziness
• tiredness
• vomiting
• stomach pain
• diarrhea
• ear pain
• runny nose
• joint pain
• dry skin

Vosoritide may cause other side effects. Call your doctor if you have any unusual problems while taking this medication.

If you experience a serious side effect, you or your doctor may send a report to the Food and Drug Administration's (FDA) MedWatch Adverse Event Reporting program online (http://www.fda.gov/Safety/MedWatch) or by phone (1-800-332-1088).

What should I know about STORAGE and DISPOSAL of this medication?

Keep this medication in the container it came in, tightly closed, and out of reach of children and away from light. Store vosoritide vials and prefilled diluent syringe in the refrigerator, but do not freeze. Before use, unmixed medication can be stored at room temperature for up to 90 days. Once mixed, use it immediately. It can be stored at room temperature for up to 3 hours after mixing. Throw away any medication that is outdated or no longer needed. Talk to your pharmacist about the proper disposal of your medication.

It is important to keep all medication out of sight and reach of children as many containers (such as weekly pill minders and those for eye drops, creams, patches, and inhalers) are not child-resistant and young children can open them easily. To protect young children from poisoning, always lock safety caps and immediately place the medication in a safe location – one that is up and away and out of their sight and reach. http://www.upandaway.org

Unneeded medications should be disposed of in special ways to ensure that pets, children, and other people cannot consume them. However, you should not flush this medication down the toilet. Instead, the best way to dispose of your medication is through a medicine take-back program. Talk to your pharmacist or contact your local garbage/recycling department to learn about take-back programs in your community. See the FDA's Safe Disposal of Medicines website (http://goo.gl/c4Rm4p) for more information if you do not have access to a take-back program.

What should I do in case of OVERDOSE?

In case of overdose, call the poison control helpline at 1-800-222-1222. Information is also available online at https://www.poisonhelp.org/help. If the victim has collapsed, had a seizure, has trouble breathing, or can't be awakened, immediately call emergency services at 911.

What OTHER INFORMATION should I know?

Keep all appointments with your doctor.

It is important for you to keep a written list of all of the prescription and nonprescription (over-the-counter) medicines you are taking, as well as any products such as vitamins, minerals, or other dietary supplements. You should bring this list with you each time you visit a doctor or if you are admitted to a hospital. It is also important information to carry with you in case of emergencies.