Voxzogo - patient leaflet, side effects, dosage

- If you or your child gets any side effects, talk to your doctor. This includes any possible side effects not listed in this leaflet. See section 4.

What is in this leaflet

• 1. What Voxzogo is and what it is used for

• 2. What you need to know before you use Voxzogo

• 3. How to use Voxzogo

• 4. Possible side effects

• 5. How to store Voxzogo

• 6. Contents of the pack and other information

1. What Voxzogo is and what it is used for

What Voxzogo is

Voxzogo contains the active substance vosoritide. It is similar to a protein in the body called C type natriuretic peptide (CNP). Vosoritide is made by recombinant technology involving bacteria that have been modified to include the gene for producing the protein.

What Voxzogo is used for

This medicine is used for the treatment of achondroplasia in patients 2 years and older whose bones are still growing. Achondroplasia is a genetic condition that affects growth of almost all bones in the body including the skull, spine, arms and legs resulting in very short stature with a characteristic appearance.

The product is indicated only in achondroplasia which is caused by mutations in the FGFR3-gene as confirmed by genetic testing.

How does Voxzogo work

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

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 using Voxzogo:

• – If you or your child have significant heart disease or blood pressure problems.

• – If you or your child are taking or have recently taken medicines that lower blood pressure.

If any of these applies to you or your child or you are unsure, talk to the doctor before using Voxzogo.

Blood pressure effects

Voxzogo can lower blood pressure. As a result, you may feel dizzy, nauseous, or tired. Blood pressure usually returns to normal within 90 minutes of 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. It is recommended patients eat a light snack and drink a glass of fluid (e.g. water, milk or juice.) about 30 minutes before injection.

Children and adolescents

There is not enough information on the use of this medicine in children under 2 years and therefore it 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 breast-feeding

If you or your child are being treated with this medicine and are pregnant or breast-feeding, think you may be pregnant or are planning to have a baby, ask your doctor for advice before using this medicine.

The use of this medicine is not recommended during pregnancy and breast-feeding.

Driving, cycling and using machines

This medicine may cause you to feel dizzy, light-headed, tired or you may feel sick shortly after your injection. If this happens, you should not drive, ride a bicycle, do physical activities or use machines for around an hour after injection or until you feel better.

Voxzogo contains sodium

This medicine contains less than 1 mmol sodium (23 mg) per dose, that is to say essentially ‘sodium-free’.

3. How to use Voxzogo

A caregiver should give the Voxzogo injection. Do not inject your child with Voxzogo until you have had proper training from a healthcare professional.

Always use this medicine exactly as your doctor has told you. Check with your doctor or pharmacist if you are not sure.

Dose

Your doctor will choose the correct dose depending on your or your child’s bodyweight. The doctor will tell you how much of the injection solution to inject. If you are not sure, ask your doctor or pharmacist.

You or your child should eat a light snack and drink a glass of water, milk or juice about 30 minutes before the injection. This can reduce side effects such as dizziness, tiredness or nausea (feeling sick).

How to use Voxzogo

Inject Voxzogo slowly under the skin (subcutaneous injection).

The injection should be given at around the same time each day.

It is recommended that you give the injection in a different place each day and do not use the same site 2 days in a row. Do not inject this medicine into moles, scars, birthmarks, or areas where the skin is tender, bruised, red, or hard.

If you use more Voxzogo than you should

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

If you forget to use Voxzogo

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

If you stop using Voxzogo

Always talk to your child’s doctor before deciding to stop your child’s treatment. 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

These may affect more than 1 in 10 people:

• Vomiting
• Low blood pressure (temporary effects include dizziness, feeling tired or feeling sick shortly after an injection)
• Injection site reactions: redness, itching, inflammation, swelling, bruising, rash, hives, pain. Injection site reactions are usually minor and resolve on their own within a few hours.

Common side effects

These may affect up to 1 in 10 people:

• Nausea
• Feeling faint or light-headed and passing out
• Dizziness
• Tiredness
• High levels of blood alkaline phosphatase (shown in blood tests)

Reporting of side effects

If you or your child get 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 inBy reporting side effects you can help provide more information on the safety of this medicine.

5. How to store Voxzogo

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

Do not use this medicine after the expiry date which is stated on the carton after “EXP”. The expiry date refers to the last day of that month.

Store in a refrigerator (2 °C – 8 °C). Do not freeze. Store in the original package in order to protect from light.

Voxzogo may be stored at room temperature (below 30 °C) for up to 90 days, but not beyond the expiry date. Do not return Voxzogo to refrigerator after storage at room temperature. Record on the carton the date you remove Voxzogo from the refrigerator to store at room temperature.

Use Voxzogo as soon as it has been made up as a solution. In any case it must be given within 3 hours of making it up. Do not use this medicine if the solution for injection is cloudy or contains any particles.

Do not throw away any medicines via household waste. Ask your pharmacist how to throw away medicines you no longer use. These measures will help protect the environment.

6. Contents of the pack and other information

What Voxzogo contains

• – The active substance is vosoritide.

• Each vial of 0.4 mg powder reconstituted in a solution of 0.5 mL solvent, corresponds to

a concentration of 0.8 mg/mL.

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

• – The other ingredients are citric acid (E 330), sodium citrate (E 331), trehalose dihydrate, mannitol (E 421), methionine, polysorbate 80 (E 433).

• – The solvent is water for injections.

What Voxzogo looks like and contents of the pack

Voxzogo powder and solvent for solution for injection is provided as:

• a white to yellow powder for solution for injection in a glass vial, and
• a clear and colourless solvent (water for injections) to dissolve the powder.

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

Each carton contains:

• 10 vials of Voxzogo
• 10 pre-filled syringes of water for injections
• 10 individual single use needles
• 10 individual single use syringes

Marketing Authorisation Holder and Manufacturer

BioMarin International Limited

Shanbally, Ringaskiddy

County Cork

Ireland

P43 R298

This leaflet was last revised in MM/YYYY.

Detailed information on this medicine is available on the European Medicines Agency web site:

. There are also links to other websites about rare diseases and treatments.

Instructions for use

Items provided to inject Voxzogo (see Figure A)

Figure A

Voxzogo vial

PREPARING FOR INJECTION

Before you start, make sure you have a clean work surface and that you have washed your hands.

Step 1: On a clean flat surface, flip off the vial cap and wipe the top with an alcohol pad. Do not touch the vial stopper with your fingers after wiping it with an alcohol pad.
Step 2: Gently bend to snap off the cap from the solvent syringe.			—\ \
Step 3: Twist the solvent needle onto the solvent syringe until you can no longer twist it.	_ »d ^
Step 4: Pull off the needle cap and insert the needle into the vial through the middle of the vial stopper. Slowly push the plunger rod down to inject all of the liquid. Be careful not to push the blue tab until Step 5.	ti -11I J		/©\j aS / /1

Step 5: Remove the needle from the vial, then press the blue tab for the needle to pull back (retract). Throw away the needle and syringe into a sharps container. See step 19 and “How to throw away (dispose of) Voxzogo.” Do not use the solvent syringe to give the injection. A ATTENTION: Be careful not to touch the needle tip.	\ 11 J I )
Step 6: Gently swirl the vial until the powder has completely dissolved and the solution is clear.			/
Do not shake.			\ I
Make sure medicine is clear to yellow, not cloudy and is particle-free.
Step 7: Pull off the needle cap from the injection syringe and insert the needle into the vial straight through the middle of the vial stopper.		—	-i-4_
Be careful not to bend the needle.	t: i □
A ATTENTION: Do not place the cap back on		1
the needle.		II! I ■■		__*
			s 1 / >
			U0 <*
		=

GIVING VOXZOGO INJECTION

Step 15: After wiping the site with an alcohol pad, pinch the skin up around the selected injection site.
	——---
Step 16: Quickly insert the needle all the way into the skin at a 45-degree angle.	______ ■
Step 17: Release the pinch and slowly push the plunger rod all the way. Inject full dose.
Step 18: Continue pressing the plunger rod until the needle retracts into the syringe.			Jr * 1 f
			—

After injecting Voxzogo

• Check the injection site. If there is a small amount of blood at the injection site, gently press a gauze pad on it for a few seconds or apply a bandage.
• Do not rub the injection site.
• Look out for signs of low blood pressure, such as dizziness, tiredness, or feeling sick. If you have these symptoms call your doctor or healthcare provider, then lie down on your back and place cushions under your legs to raise them.

How to throw away (dispose of) Voxzogo

Put your used or expired vials, needles and syringes in a sharps disposal container right away after use.

If you do not have a 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 labelled to warn of hazardous waste inside the container