VOXZOGO 0.56 mg POWDER AND SOLVENT FOR INJECTABLE SOLUTION

Introduction

Package Leaflet: Information for the User

Voxzogo 0.4 mg powder and solvent for solution for injection

Voxzogo 0.56 mg powder and solvent for solution for injection

Voxzogo 1.2 mg powder and solvent for solution for injection

vosoritide

This medicinal product is subject to additional monitoring, which will allow for the quick identification of new safety information. You can help by reporting any side effects you may have to your doctor. The last section of section 4 will tell you how to report side effects.

Read all of this leaflet carefully before you start using this medicine, because it contains important information for you.

• Keep this leaflet, you may need to read it again.
• If you or your child have any further questions, ask your doctor or pharmacist.
• This medicine has been prescribed for you or your child only. Do not pass it on to others. It may harm them, even if their signs of illness are the same as yours.
• If you or your child get any side effects, talk to your doctor or pharmacist. This includes any possible side effects not listed in this leaflet. See section 4.

Contents of the pack

1. What is Voxzogo and what is it used for
2. What you need to know before you use Voxzogo
3. How to use Voxzogo
4. Possible side effects
5. Storage of Voxzogo
6. Contents of the pack and other information

1. What is Voxzogo and what is it used for

What is Voxzogo

Voxzogo contains the active substance vosoritide. It is similar to a protein found in the body called C-type natriuretic peptide (CNP). Vosoritide is made using recombinant technology, where bacteria are modified to include the gene that produces the protein.

What Voxzogo is used for

This medicine is used to treat achondroplasia in patients aged 4 months and older whose bones are still growing. Achondroplasia is a genetic disorder that affects the growth of almost all the bones in the body, including the bones of the skull, spine, arms, and legs, resulting in a greatly reduced height with a characteristic appearance.

This product is only indicated for achondroplasia caused by a mutation in the FGFR3 gene, confirmed by genetic testing.

How Voxzogo works

The active substance in Voxzogo works directly on the growth plates of the bones to promote the growth of new bone.

2. What you need to know before you use Voxzogo

Do not use Voxzogo

• if you or your child are allergic to vosoritide or any of the other ingredients of this medicine (listed in section 6).

Warnings and precautions

Talk to your doctor before starting Voxzogo:

• if you or your child have significant heart or blood pressure problems;
• if you or your child are taking or have recently taken medicines to lower blood pressure.

If any of the above applies to you or your child, or if you are not sure, talk to your doctor before using Voxzogo.

Effects on blood pressure

Voxzogo may lower blood pressure. As a result, you may feel dizzy, tired, or nauseous. Blood pressure usually returns to normal within 90 minutes after the Voxzogo injection. If these effects occur and are severe, tell your doctor.

Drinking plenty of fluids at the time of injection may reduce the likelihood of these effects. Patients are recommended to eat a snack and drink enough fluid (such as water, milk, or juice) about 30 minutes before the injection.

Children and adolescents

There is not enough information on the use of this medicine in children under 4 months, and therefore, its use is not recommended.

Other medicines and Voxzogo

Tell your doctor if you or your child are taking, have recently taken, or might take any other medicines.

Pregnancy and breastfeeding

If you or your daughter are receiving treatment with this medicine and you or your daughter are pregnant or breastfeeding, think you may be pregnant, or plan to become pregnant, consult your doctor before using this medicine.

The use of this medicine is not recommended during pregnancy and breastfeeding.

Driving, cycling, and using machines

This medicine may make you feel dizzy, tired, or nauseous for a few minutes after the injection. In this case, do not drive, cycle, engage in physical activities, or use machines for about 1 hour after the injection or until you feel better.

Voxzogo contains sodium

This medicine contains less than 1 mmol of sodium (23 mg) per dose; this is essentially "sodium-free".

3. How to use Voxzogo

The Voxzogo injection should be given by a caregiver. Do not inject Voxzogo into your child until you have received the necessary training from a healthcare professional.

Follow the instructions for administering this medicine exactly as told by your doctor. If you are unsure, consult your doctor or pharmacist again.

Dose

Your doctor will choose the correct dose based on your or your child's body weight. The doctor will tell you how much solution for injection to inject. If you are unsure, consult your doctor or pharmacist.

Table 1 shows the dose that you or your child will need to inject daily based on body weight. The amount to inject will be presented in different volumes depending on the type of syringe included in the pack (milliliters (ml) or units (U)). Check that you have the correct dose for the syringe you are using.

Table 1: Single-dose volumes by body weight in ml and unit volumes (U)

You or your child should eat a snack and drink enough water, milk, or juice about 30 minutes before receiving the injection. This may reduce side effects such as dizziness, tiredness, or nausea.

How to use Voxzogo

Inject Voxzogo slowly under the skin (subcutaneous injection).

The injection should be given at about the same time every day.

It is recommended to inject at a different site each day and not to use the same site 2 days in a row. Do not inject the medicine into areas with moles, scars, or birthmarks, or into areas where the skin is sensitive, bruised, red, or hard.

If you use more Voxzogo than you should

If you inject more Voxzogo than you should, contact your doctor immediately.

If you forget to use Voxzogo

If your child misses a dose, they should be given the injection if it is not more than 12 hours since the scheduled time. If more than 12 hours have passed since the scheduled time, the missed dose should not be given. Wait until the next day and continue with the usual dose at the usual time.

If you stop using Voxzogo

Before deciding to stop your child's treatment, always consult your child's doctor. If you or your child have any further questions on the use of this medicine, ask your doctor or pharmacist.

4. Possible side effects

Like all medicines, this medicine can cause side effects, although not everybody gets them.

Very common side effects

May affect more than 1 in 10 people:

• Vomiting
• Low blood pressure (temporary effects include dizziness, tiredness, or nausea shortly after receiving the injection)
• Injection site reactions: redness, itching, inflammation, swelling, bruising, rash, hives, pain. Injection site reactions are usually mild and resolve on their own within a few hours.
• High levels of alkaline phosphatase in the blood (shown in blood tests)

Common side effects

May affect up to 1 in 10 people:

• Nausea
• Feeling of dizziness and loss of consciousness
• Dizziness
• Tiredness

Reporting of side effects

If you or your child experience any side effects, talk to your doctor or nurse. This includes any possible side effects not listed in this leaflet. You can also report side effects directly via the national reporting system listed in Appendix V. By reporting side effects, you can help provide more information on the safety of this medicine.

5. Storage of Voxzogo

Keep this medicine out of the sight and reach of children.

Do notuse this medicine after the expiry date which is stated on the carton after "EXP". The expiry date is the last day of the month shown.

Store in a refrigerator (between 2°C and 8°C). Do not freeze.Keep the pack in the outer carton in order to protect from light.

Voxzogo may be stored at room temperature (below 30°C) for up to 90 days, but not after the expiry date. Do notreturn Voxzogo to the refrigerator after it has been stored at room temperature. Writethe date on the carton when you remove Voxzogo from the refrigerator to store at room temperature.

Use Voxzogo as soon as it is prepared as a solution. In any case, it must be administered within 3 hours of preparation. Do not use this medicine if the solution for injection is cloudy or contains particles.

Medicines should not be disposed of via wastewater or household waste. Ask your pharmacist how to dispose of medicines no longer required. This will help protect the environment.

6. Container Contents and Additional Information

Voxzogo Composition

• The active ingredient is vosoritide.

• Each 0.4 mg vial of reconstituted powder in a 0.5 ml solvent solution corresponds to a concentration of 0.8 mg/ml.
• Each 0.56 mg vial of reconstituted powder in a 0.7 ml solvent solution corresponds to a concentration of 0.8 mg/ml.
• Each 1.2 mg vial of reconstituted powder in a 0.6 ml solvent solution corresponds to a concentration of 2 mg/ml.

• The other components are citric acid (E 330), sodium citrate (E 331), trehalose dihydrate, mannitol (E 421), methionine, and polysorbate 80 (E 433).
• The solvent is water for injectable preparations.

Appearance of Voxzogo and Container Contents

Voxzogo powder and solvent for injectable solution are supplied as:

• a powder for injectable solution that is white to yellow in color in a glass vial and
• a clear and colorless solvent (water for injectable preparations) to dissolve the powder.

After dissolving the powder in the solvent, the solution is a clear liquid, colorless to yellow.

Each box contains:

• 10 Voxzogo vials
• 10 pre-filled syringes with water for injectable preparations
• 10 single-use needles
• 10 single-use syringes

Marketing Authorization Holder and Manufacturer

BioMarin International Limited

Shanbally, Ringaskiddy

County Cork

Ireland

P43 R298

Date of Last Revision of this Leaflet: MM/YYYY.

Detailed information on this medicinal product is available on the European Medicines Agency website: http://www.ema.europa.eu. There are also links to other websites on rare diseases and orphan medicines.

Instructions for Use of the Graduated Syringe in Milliliters (ml)

Read these instructions for use before using Voxzogo and every time you perform a refill. There may be new information.

Supplied Elements for Injecting Voxzogo (see Figure A)

Figure A

Injectable Syringe

Consult your doctor or healthcare professional if you are unsure of the recommended dose or how to use the solvent needle and injectable syringe.

Necessary Elements Not Supplied in the Container (see Figure B)

If you do not have these elements, consult your pharmacist.

Figure B

PREPARATION FOR INJECTION

Before starting, make sure the work surface is clean and that you have washed your hands.

SELECTION AND PREPARATION OF THE INJECTION SITE

ADMINISTRATION OF THE VOXZOGO INJECTION

AFTER INJECTING VOXZOGO

• Check the injection site. If you see a small amount of blood at the injection site, press gently with a gauze for a few seconds or apply a dressing.
• Do notrub the injection site.
• Be aware of signs of low blood pressure, such as dizziness, fatigue, or feeling unwell. If you have these symptoms, call your doctor or healthcare professional and then lie down on your back and put pillows under your legs to keep them elevated.

HOW TO DISCARD (DISPOSE OF) VOXZOGO

Place used or expired vials, needles, and syringes in a sharps container immediately after use.

If you do not have a sharps container, you can use a household container that:

• is made of heavy-duty plastic;
• can be closed with a tight-fitting, puncture-resistant lid that prevents sharps from coming out;
• is kept upright and stable during use;
• is leak-resistant and
• is properly labeled to warn of hazardous waste.

When the container you use for disposing of sharps is almost full, you must follow local guidelines for disposing of it properly.

Medicines, vials, loose needles, and syringes should not be thrown away in the trash. Ask your pharmacist how to dispose of the packaging and medicines you no longer need. This will help protect the environment.

INSTRUCTIONS FOR USE OF THE GRADUATED SYRINGE IN UNITS (U)

The solvent needles and administration syringes included in this package are new components marked with “Units” (U) to measure the recommended dose. Your doctor will indicate the recommended dose to administer based on your weight interval.

Supplied Elements for Injecting Voxzogo (see Figure A)

Figure A

Injectable Syringe

Your dose can be administered using the injectable syringe in Figure A. The measurements for this syringe are equivalent to ml as follows: 0.1 ml = 10 units.

Necessary Elements Not Supplied in the Container (see Figure B)

If you do not have these elements, consult your pharmacist.

Figure B

PREPARATION FOR INJECTION

Before starting, make sure the work surface is clean and that you have washed your hands.

SELECTION AND PREPARATION OF THE INJECTION SITE

ADMINISTRATION OF THE VOXZOGO INJECTION

AFTER INJECTING VOXZOGO

• Check the injection site. If you see a small amount of blood at the injection site, gently press with a gauze for a few seconds or apply a bandage.
• Do notrub the injection site.
• Be aware of signs of low blood pressure, such as dizziness, fatigue, or feeling unwell. If you have these symptoms, call your doctor or healthcare professional and then lie down on your back and place pillows under your legs to keep them elevated.

HOW TO DISPOSE OF (DISCARD) VOXZOGO

Place used or expired vials, needles, and syringes in a sharps container immediately after use.

If you do not have a sharps container, you can use a household container that:

• is made of heavy-duty plastic;
• can be closed with a tight-fitting, puncture-resistant lid that prevents the sharps from coming out;
• is kept upright and stable during use;
• is leak-resistant and
• is properly labeled to warn that it contains hazardous waste.

When the container you use to discard sharps is almost full, you should follow local guidelines for proper disposal.

Medications, vials, loose needles, and syringes should not be thrown away in the trash. Ask your pharmacist how to dispose of containers and medications you no longer need. This will help protect the environment.