Prospect: information for the user

Voxzogo 0.4 mg powder and solvent for injectable solution

Voxzogo 0.56 mg powder and solvent for injectable solution

Voxzogo 1.2 mg powder and solvent for injectable solution

vosoritida

This medicine is subject to additional monitoring, which will speed up the detection of new information about its safety. You can contribute by reporting any adverse effects that you or your child may experience. The final part of section 4 includes information on how to report these adverse effects.

Read this prospect carefully before starting to use this medicine, because it contains important information for you.

• Keep this prospect, as you may need to read it again.
• If you or your child have any doubts, consult your doctor.
• This medicine has been prescribed only to you or your child, and should not be given to other people even if they have the same symptoms as you, as it may harm them.
• If you or your child experience any adverse effects, consult your doctor, even if they are possible adverse effects that do not appear in this prospect. See section 4.

What is Voxzogo

Voxzogo contains the active ingredient vosoritida. It is similar to a protein present in the body called C-type natriuretic peptide (CNP). Vosoritida is produced using recombinant technology in which modified bacteria are used that include the gene that produces the protein.

How is Voxzogo used

This medication is used for the treatment of achondroplasia in patients 4 months of age and older whose bones are still growing. Achondroplasia is a genetic disease 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 significantly reduced stature with a characteristic appearance.

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

How Voxzogo works

The active ingredient of Voxzogo acts directly on the growth points of the bones to promote the growth of new bone.

Do not use Voxzogo

• if you or your child are allergic to vosoritida or any of the other components of this medication (listed in section 6).

Warnings and precautions

Consult your doctor before starting to use Voxzogo:

• if you or your child have significant heart disease or high blood pressure issues;
• if you or your child are taking or have recently taken medications to lower blood pressure.

If any of the above cases apply to you or your child or if you have doubts, consult your doctor before using Voxzogo.

Effects on blood pressure

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

Drinking plenty of liquids at the time of the injection may reduce the likelihood of these effects. It is recommended that patients eat a snack and drink sufficient liquid (such as water, milk, or juice) approximately 30 minutes before the injection.

Children and adolescents

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

Other medications and Voxzogo

Inform your doctor if you or your child are taking, have recently taken, or may need to take any other medication.

Pregnancy and breastfeeding

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

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

Driving, cycling, and using machines

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

Voxzogo contains sodium

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

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

Follow exactly the administration instructions for this medication as indicated by your doctor. If in doubt, consult your doctor or pharmacist again.

Dose

Your doctor will choose the correct dose according to your body weight or your child's body weight. The doctor will tell you how much injectable solution to inject. If in doubt, consult your doctor or pharmacist.

The table 1 shows the dose that you or your child will have to inject daily according to body weight. The amount to be injected will be presented in different volumes according to the type of syringe included in your package (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 (U) volumes

You or your child should eat a snack and drink enough water, milk or juice approximately 30 minutes before receiving the injection. This may reduce side effects, such as dizziness, fatigue or nausea (sensation of illness).

How to use Voxzogo

Inject Voxzogo slowly under the skin (subcutaneous injection).

The injection should be administered approximately at the same time every day.

It is recommended to administer the injection in a different place each day and not to use the same place 2 days in a row. Do not inject the medication in areas with moles, scars or birthmarks, or in areas where the skin is sensitive, purple, 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, the injection should be administered if it has not been more than 12 hours since the scheduled time. If it has been more than 12 hours since the scheduled administration time, the missed dose should not be administered. Wait until the next day and continue with the usual dose at the usual time.

If you interrupt the treatment with Voxzogo

Before deciding to interrupt the treatment of your child, consult your child's doctor. If you or your child have any other questions about the use of this medication, ask your doctor or pharmacist.

Like all medications, this medication may produce adverse effects, although not all people will experience them.

Very Frequent Adverse Effects

May affectmore than 1 in 10 people:

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

Frequent Adverse Effects

May affectup to 1 in 10 people:

• Nausea
• Sensation of dizziness and loss of consciousness
• Dizziness
• Fatigue

Reporting Adverse Effects

If you or your child experience any type of adverse effect, consult your doctor or nurse, even if it is a possible adverse effect not listed in this prospectus. You can also report them directly through thenational notification system included in theAppendix V. By reporting adverse effects, you can contribute to providing more information about the safety of this medication.

Keep this medicationout of the sight and reach of children.

Do notuse this medication after the expiration date that appears on the box after «CAD». The expiration date is the last day of the month indicated.

Store in the refrigerator (between 2 °C and 8 °C).Do not freeze.Store in the original packaging to protect it from light.

Voxzogo can be stored at room temperature (less than 30 °C) for up to 90 consecutive days, but not after the expiration date.Do notreturn Voxzogo to the refrigerator after storing it at room temperature.Markin the boxthe datewhen you removed Voxzogo from the refrigerator to store it at room temperature.

Use Voxzogo as soon as it has been prepared in solution form. In any case, it must be administered within 3 hours of preparation. Do not use this medication if the injectable solution is cloudy or contains particles.

Medicines should not be disposed of through drains or in the trash. Ask your pharmacist how to dispose of the containers and medications that you no longer need. In this way, you will help protect the environment.

Composition of Voxzogo

• The active ingredient is vosoritida.

• Each vial of 0.4 mg of powder reconstituted in a solution of 0.5 ml of solvent corresponds to a concentration of 0.8 mg/ml.
• Each vial of 0.56 mg of powder reconstituted in a solution of 0.7 ml of solvent corresponds to a concentration of 0.8 mg/ml.
• Each vial of 1.2 mg of powder reconstituted in a solution of 0.6 ml of solvent 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 polisorbate 80 (E 433).
• The solvent is water for injection.

Appearance of Voxzogo and contents of the package

Voxzogo powder and solvent for injection solution are supplied as:

• a white to yellow powder for injection solution in a glass vial and
• a transparent and colorless (water for injection) solvent to dissolve the powder.

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

Each box contains:

• 10 vials of Voxzogo
• 10 preloaded syringes with water for injection
• 10 individual single-use needles
• 10 individual single-use syringes

Marketing authorization holder and responsible manufacturer

BioMarin International Limited

Shanbally, Ringaskiddy

County Cork

Ireland

P43 R298

Last review date of this leaflet: MM/AAAA.

The detailed information on this medicine 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 each time you refill. There may be new information.

Elements supplied for injecting Voxzogo (see Figure A)

Figure A

Syringe for injectables

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

Elements necessary but not supplied in the package (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 your hands have been washed.

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 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 and place pillows under your legs to keep them elevated.

How to dispose of (eliminate) Voxzogo

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

If you do not have a sharp objects container, you can use a domestic container that:

• is made of resistant plastic;
• can be closed with a tight-fitting and puncture-resistant lid that prevents the sharp objects from coming out;
• remains in an upright and stable position during use;
• is resistant to leaks and
• is properly labeled to warn that it contains hazardous waste.

When the container you use to dispose of sharp objects is almost full, you must follow local guidelines for proper disposal.

Medicines, vials, loose needles, and syringes should not be thrown away in the trash. Ask your pharmacist how to dispose of empty containers and unused medicines. This will help protect the environment.

Instructions for use of the graduated syringe in units (U)

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

Elements supplied for injecting Voxzogo (see Figure A)

Figure A

Syringe for injectables

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

Elements necessary but not supplied in the package (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 your hands have been washed.

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 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 and place pillows under your legs to keep them elevated.

How to dispose of (eliminate) Voxzogo

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

If you do not have a sharp objects container, you can use a domestic container that:

• is made of resistant plastic;
• can be closed with a tight-fitting and puncture-resistant lid that prevents the sharp objects from coming out;
• remains in an upright and stable position during use;
• is resistant to leaks and
• is properly labeled to warn that it contains hazardous waste.

When the container you use to dispose of sharp objects is almost full, you must follow local guidelines for proper disposal.

Medicines, vials, loose needles, and syringes should not be thrown away in the trash. Ask your pharmacist how to dispose of empty containers and unused medicines. This will help protect the environment.