Voxzogo 1.2 mg powder and solvent for solution for injection

Spain
Brand name Voxzogo 1.2 mg powder and solvent for solution for injection
Form powder and solution for injection
Active substance / Dosage
VOSORITIDE · 1,2 mg
Prescription type Hospital Diagnosis
ATC code
M05B M05BX M05BX07
Registration number 1211577003
Manufacturer Biomarin International Limited

Table of Contents

Package leaflet: Information for the user

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 faster detection of new safety information. You can help by reporting any adverse reactions that you or your child may experience. Information on how to report adverse reactions is included at the end of section 4.

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

  • Keep this leaflet, as you may need to read it again.
  • If you or your child have any questions, consult your doctor.
  • This medicine has been prescribed only for you or your child and must not be given to others, even if they have the same symptoms as you, because it may harm them.
  • If you or your child experience any adverse reactions, consult your doctor, even if they are possible adverse reactions not listed in this leaflet. See section 4.

Leaflet contents

  1. What Voxzogo is and what it is used for
  2. What you need to know before using Voxzogo
  3. How to use Voxzogo
  4. Possible adverse 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 naturally present in the body called C-type natriuretic peptide (CNP). Vosoritide is produced using recombinant technology, in which modified bacteria are used to incorporate the gene responsible for producing the protein.

What Voxzogo is used for

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

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

How Voxzogo works

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

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

Do not use Voxzogo

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

Warnings and precautions

Talk to your doctor before starting to use Voxzogo:

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

If any of the above apply to you or your child, or if you have any 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. Usually, blood pressure returns to normal within 90 minutes after the Voxzogo injection. If these effects occur and are severe, inform your doctor.

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

Children and adolescents

There is insufficient information on the use of this medicine in children under 4 months of age, 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 need to take any other medicines.

Pregnancy and breastfeeding

If you or your daughter are being treated with this medicine and you or your daughter are pregnant or breastfeeding, think you might be pregnant, or plan to become pregnant, consult your doctor before using this medicine.

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

Driving, cycling, and using machines

This medicine may make you feel dizzy, tired, or nauseous for a few minutes after receiving the injection. In this case, you should not drive, cycle, engage in physical activities, or operate machinery for approximately 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; essentially “sodium-free”.

3. How to use Voxzogo

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

Always follow exactly the instructions for administration provided by your doctor. If in doubt, consult your doctor or pharmacist again.

Dosage

Your doctor will select the correct dose based on your body weight or that of your child. Your doctor will tell you how much injectable solution to administer. If in doubt, consult your doctor or pharmacist.

Table 1 shows the daily dose to be injected according to body weight. The amount to be injected will be presented in different volumes depending on the type of syringe included in your package (milliliters (ml) or units (U)). Make sure you have the correct dose for the syringe you are using.

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

Body

weight

(kg)

Dose (mg)

Vosoritide 0,4 mg

solvent (water for

injectable

preparations): 0.5 ml

concentration:

0.8 mg/ml

Vosoritide 0.56 mg

solvent (water for

injectable

preparations): 0.7 ml

concentration:

0.8 mg/ml

Vosoritide 1.2 mg

solvent (water for

injectable

preparations): 0.6 ml

concentration: 2 mg/ml

Daily injection volume

ml

Units

ml

Units

ml

Units

4

0.12 mg

0.15 ml

15 U

5

0.16 mg

0.20 ml

20 U

6–7

0.20 mg

0.25 ml

25 U

8–11

0.24 mg

0.30 ml

30 U

12–16

0.28 mg

0.35 ml

35 U

17–21

0.32 mg

0.40 ml

40 U

22–32

0.40 mg

0.50 ml

50 U

33–43

0.50 mg

0.25 ml

25 U

44–59

0.60 mg

0.30 ml

30 U

60–89

0.70 mg

0.35 ml

35 U

≥ 90

0.80 mg

0.40 ml

40 U

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, tiredness, or nausea (feeling unwell).

How to use Voxzogo

Inject Voxzogo slowly under the skin (subcutaneous injection).

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

It is recommended to administer the injection at a different site each day and not to use the same site on two consecutive days. Do not inject the medicine into areas with moles, scars, or birthmarks, or into areas where the skin is tender, bruised, red, or hard.

If you use more Voxzogo than you should

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

If you forget to use Voxzogo

If your child misses a dose, the injection should be given if less than 12 hours have passed since the scheduled time. If more than 12 hours have passed since the scheduled time, the missed dose should not be administered. Wait until the next day and continue with the regular 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 about the use of this medicine, ask your doctor or pharmacist.

4. Possible adverse effects

Like all medicines, this medicine can cause adverse effects, although not everyone gets them.

Very common adverse effects

May affect more than 1 in 10 people:

  • Vomiting
  • Low blood pressure (temporary effects include dizziness, tiredness or feeling unwell shortly after receiving the injection)
  • Reactions at the injection site: redness, itching, swelling, puffiness, bruising, rash, 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)

Common adverse effects

May affect up to 1 in 10 people:

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

Reporting of 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 leaflet. You may also report them directly through the national reporting system included in Appendix V. By reporting adverse 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 not use this medicine after the expiry date stated on the carton after "EXP". The expiry date refers to the last day of the month indicated.

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

Voxzogo may be stored at room temperature (below 30 °C) for up to 90 consecutive days, but not beyond the expiry date. Do not return Voxzogo to the refrigerator after storing it at room temperature. Record on the carton the date when Voxzogo was removed from the refrigerator for storage at room temperature.

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

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

6. Contents of the pack and other information

Composition of Voxzogo

  • The active substance is vosoritide.

  • Each vial containing 0.4 mg of powder reconstituted in 0.5 ml of solvent corresponds to a concentration of 0.8 mg/ml.

  • Each vial containing 0.56 mg of powder reconstituted in 0.7 ml of solvent corresponds to a concentration of 0.8 mg/ml.

  • Each vial containing 1.2 mg of powder reconstituted in 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 polysorbate 80 (E 433).

  • The solvent is water for injections.

Appearance of Voxzogo and contents of the pack

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

  • a white to yellow powder for solution for injection in a glass vial, and
  • a clear, 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 with water for injections
  • 10 single-use individual needles
  • 10 single-use individual syringes

Marketing Authorisation Holder and Manufacturer

BioMarin International Limited

Shanbally, Ringaskiddy

County Cork

Ireland

P43 R298

Date of the most recent review of this leaflet: MM/YYYY.

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

Instructions for use of the syringe graduated in millilitres (ml)

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

Items supplied for injecting Voxzogo (see Figure A)

Figure A

Voxzogo vial

Solvent syringe needle

(the blue tab retracts the needle)

Solvent syringe (contains water for

injectable preparations for

reconstitution of Voxzogo)

Stylized drawing of a glass vial with cap and a circular rubber stopper, with a dotted border, on a white background

Technical diagram of a medical device with a horizontal cylindrical body and a

Black-and-white technical drawing of a horizontal syringe with plunger, graduated barrel, and tip with protective cap

Syringe for injectable use

Black-and-white technical drawing of a horizontal syringe with milliliter scale and extended plunger

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

Items required but not supplied in the package (see Figure B)

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

Figure B

Alcohol-impregnated wipes

Sharps container

Gauze or dressings

Stylized black-and-white drawing of two square tablets with rounded corners and thin black borders, overlapping each other

White container with gray lid and a black biological hazard symbol centered on a neutral background

Schematic drawing of a gray adhesive bandage applied over a dotted, tilted white square on a neutral background

PREPARATION FOR INJECTION

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

Step 1: On a clean, flat surface, remove

the closure cap from the vial and wipe the top

with an alcohol swab.

Do not touch the vial stopper with your fingers after

wiping it with an alcohol swab.

Technical drawing showing how to remove the cap from a medical vial by rotating and lifting it with fingers to access the contents

Step 2: Gently twist off the cap from the solvent syringe.

Two hands rotating and removing a gray cap from the top of a medical syringe, following a black curved arrow

Step 3: Screw the solvent needle onto the

solvent syringe until it can no longer be tightened.

Two hands attaching a protective cap to a syringe, with a black curved arrow indicating the downward insertion movement

Step 4: Remove the needle cap and insert the

needle into the vial through the center of the stopper.

Slowly push the syringe plunger to

inject all the liquid.

Be careful not to push the blue tab

until Step 5.

Technical drawing showing two stages: the

Step 5: Remove the needle from the vial and then

press the blue tab to retract the needle. Dispose of the needle and syringe in a sharps container.

See Step 19 and “How to dispose of (discard)

Voxzogo”.

Do not use the solvent syringe to administer the

injection.

CAUTION: Be careful not to touch the needle tip.

One hand holding a syringe with needle pointing downward while the

Two hands holding a syringe with a blue device and a black arrow indicating movement to the right for

Step 6: Gently swirl the vial in a circular motion until the powder is completely dissolved and the solution is clear.

Do not shake the vial.

Ensure the medication is from transparent to yellow in color and is not cloudy or containing particles.

Two hands holding a glass vial while a gray circular arrow indicates rotational movement around the container

Step 7: Remove the cap from the injection syringe needle and insert the needle straight into the vial through the center of the stopper.

Be careful not to bend the needle.

CAUTION: Do not recap the needle.

Technical drawing showing the step of removing the cap from the syringe and the

Step 8: Carefully hold the vial and syringe and invert the vial with the needle still inserted. The vial should be on top.

Be careful not to bend the needle.

Two hands holding a syringe with a medicine vial on top, highlighted by a dotted circle to indicate the

Step 9: Keep the needle tip in the

medication and slowly pull back on the plunger to draw the prescribed dose into the syringe.

Check the prescription label for the

amount to be drawn.

CAUTION: Draw up the prescribed dose.

One hand holding a syringe vertically with a finger on the plunger while the

Step 10: Gently tap the syringe with a

finger to remove large air bubbles. Then, slowly

push the bubbles back into the vial.

Two hands manipulating a syringe with a black arrow indicating movement toward the

Step 11: Repeat Steps 9 and 10 until the correct prescribed dose is in the syringe and there are no large air bubbles.

Ensure the dose in the syringe matches the prescribed dose. Measure from the base of the

plunger, as shown in the figure.

CAUTION: Remove large air bubbles. One or two small bubbles are acceptable.

Medical diagram showing two graduated scales with dosage indicators positioned between 0.5 and 0.7, and an arrow pointing to level 0.6

Step 12: Confirm the prescribed dose is in the syringe, then remove the vial and prepare to administer the dose.

CAUTION: Before removing the vial,

verify that the amount drawn matches the prescribed dose.

One hand lifting a protective cap upward toward the

CHOICE AND PREPARATION OF THE INJECTION SITE

Step 13: Voxzogo must be injected into the fatty layer under the skin (subcutaneous layer) only.

  • Do not inject through clothing.
  • Do not inject in the same area two times in a row.
  • Do not inject into skin that is painful, bruised, red, hard, or scarred.

The following areas are recommended for injections:

  • back of the upper arms or
  • thighs or
  • abdomen (5 centimeters from the navel) or
  • buttocks

Human body diagram showing injection sites with gray areas on the

Step 14: Clean the injection site with an alcohol wipe and allow the skin to air dry.

Do not touch the area again before administering the injection.

Line drawing showing a hand placing a small patch or applicator gently onto the surface of a skin area or wound

ADMINISTRATION OF VOXZOGO INJECTION

Step 15: After cleaning the area with an

alcohol wipe, pinch the skin upward at

the chosen injection site.

Line drawing showing a hand pinching and lifting a skin fold on a curved body surface

Step 16: Quickly insert the entire needle into

the skin at a 45-degree angle.

Technical drawing showing a hand inserting a needle with a graduated syringe into the skin with a

Step 17: Release the pinch and, slowly, push the

syringe plunger all the way down. Inject the

full dose.

Two hands holding a tilted syringe while the tip of the

Step 18: Continue pressing the plunger until

the needle retracts into the syringe.

Diagram showing two hands holding a syringe to inject medication into subcutaneous tissue with different angles and depths

Step 19: Dispose of the vial, syringes, and used

needles in a sharps disposal container. See “How to dispose of (discard)

Voxzogo” for more information.

Black downward arrow indicating disposal of a syringe and vial into a hazardous biological waste container

After injecting Voxzogo

  • Check the injection site. If you see a small amount of blood at the injection site, gently press with a gauze pad for a few seconds or apply a bandage.
  • Do not rub the injection site.
  • Watch for signs of low blood pressure, such as dizziness, tiredness, or feeling unwell. If you experience these symptoms, call your doctor or healthcare professional and then lie down on your back with pillows under your legs to keep them elevated.

How to dispose of Voxzogo

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

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

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

When the container you are using for sharps disposal is nearly full, follow local guidelines for proper disposal.

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

Instructions for using the syringe graduated 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 instruct you on the recommended dose to administer based on your weight range.

Items supplied for injecting Voxzogo (see Figure A)

Figure A

Voxzogo vial

Solvent syringe needle

Solvent syringe (contains water for

injectable preparations for reconstitution

of Voxzogo)

Stylized drawing of a medicine vial next to a top-down view of a

Black-and-white technical drawing of a syringe-shaped medical device or autoinjector with a cylindrical body and shaped distal end

Black-and-white technical drawing of a horizontal syringe with plunger, graduated barrel, and tip with protective cap

Injectable syringe

Horizontal syringe with a scale numbered from 10 to 100 and measurement marks for dosing liquid medication

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

Items required but not supplied in the package (see Figure B)

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

Figure B

Alcohol-impregnated wipes

alcohol

Sharps container

for sharp objects

Gauze or dressings

Stylized black-and-white drawing of two square tablets with rounded corners and thin black borders, overlapping each other

Gray icon of a sharps container with a white circle containing a black syringe symbol and the text 'Objetos Cortopunzantes'

Schematic drawing of a gray adhesive bandage applied on a dotted light skin surface with small white perforations

PREPARATION FOR INJECTION

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

Step 1: On a clean, flat surface, remove the cap from the vial and clean the top with an alcohol wipe.

Do not touch the vial stopper with your fingers after

cleaning it with the alcohol wipe.

Schematic drawing showing a hand lifting the rubber stopper from a medicine vial, with a curved arrow indicating the movement

Step 2: Gently twist off the cap of the solvent syringe.

Two hands rotating and removing a gray cap from a syringe, with a black curved arrow indicating the rotational movement

Step 3: Screw the solvent needle onto the solvent syringe until it can no longer be turned.

Schematic drawing showing two hands separating a medical device into two parts, with a curved arrow indicating the detachment movement between components

Step 4: Remove the cap from the needle and insert the needle into the vial through the center of the stopper. Slowly push the plunger of the syringe to inject all the liquid.

Technical drawing showing hands extracting the

Step 5: Remove the needle from the vial. Dispose of the needle and syringe in a sharps container. See Step 18 and “How to dispose of (discard) Voxzogo.”

Do not use the solvent syringe to administer the

injection.

CAUTION: Be careful not to touch the tip of the needle.

Technical drawing showing two stages: one hand lifting a syringe from a vial and the

Step 6: Gently swirl the vial in a circular motion until the powder is completely dissolved and the solution is clear.

Do not shake the vial.

Ensure the medication is colorless to yellow and is not cloudy or containing particles.

One hand holding a glass vial while a gray circular arrow indicates a rotational movement around the container

Step 7: Remove the cap from the injection syringe needle and insert the needle straight into the vial through the center of the stopper.

Be careful not to bend the needle.

CAUTION: Do not recap the needle.

Diagram showing movement toward the

Step 8: Carefully hold the vial and syringe and invert the vial with the needle still inserted. The vial should be on top.

Be careful not to bend the needle.

Diagram showing two hands holding a vertical syringe to prepare the dosage, with a dotted circle highlighting the upper part of the container

Step 9: Keep the tip of the needle in the medication and slowly pull back the plunger to withdraw the prescribed dose into the syringe.

Check the prescription label for the amount to be withdrawn.

CAUTION: Check the syringe supplied in the package and withdraw the prescribed dose.

One hand holding a syringe vertically with internal liquid while the

Step 10: Gently tap the syringe with a finger to remove large air bubbles. Then, slowly push the air bubbles back into the vial.

Two hands gripping a sy游戏副本 with a finger pressing the plunger downward while a black arrow indicates the movement toward the

Step 11: Repeat Steps 9 and 10 until the correct prescribed dose is in the syringe and there are no large air bubbles.

Ensure the dose in the syringe matches the prescribed dose. Measure from the base of the plunger, as shown in the figure.

CAUTION: Remove large air bubbles. One or two small bubbles are acceptable.

Diagram showing the syringe plunger positioned between the 60 and 70 marks on a graduated scale with reference arrows

Step 12: Confirm that you have the prescribed dose in the syringe, then remove the vial and prepare to administer the dose.

CAUTION: Before removing the vial, verify that the amount withdrawn matches the prescribed dose.

One hand lifting a protective cap upward toward the

SELECTION AND PREPARATION OF THE INJECTION SITE

Step 13: Voxzogo must be injected into the fatty layer beneath the skin (subcutaneous layer)

only.

  • Do not inject through clothing.
  • Do not inject into the same site twice in a row.
  • Do not inject into skin that is painful, bruised, red, hard, or scarred.

The following areas are recommended for injections:

  • back of the upper arms or
  • thighs or
  • abdomen (5 centimeters from the navel) or
  • buttocks

One hand holding and pressing a small tablet or medical disc onto a flat, slightly curved surface

Step 14: Clean the injection site with an

alcohol wipe and allow the skin to dry in the

air.

Do not touch the area again before administering

the injection.

One hand pinching the skin of the arm or thigh, creating a fold to prepare for

ADMINISTRATION OF VOXZOGO INJECTION

Step 15: After cleaning the area with an

alcohol wipe, pinch the skin upward at

the chosen injection site.

One hand pinching the skin of the arm or thigh, creating a fold to prepare for

Step 16: Quickly insert the entire needle into

the skin at a 45-degree angle.

One hand holding a syringe tilted at 45 degrees while the

Step 17: Release the pinch and, slowly, push the

syringe plunger all the way down. Inject the

full dose.

Two hands holding a tilted syringe to insert the

Step 18: Dispose of the vial, syringes, and used

needles in a sharps container. See “How to dispose

of (discard) Voxzogo” for more information.

Schematic drawing of a syringe and needle being placed into a biological waste container marked with the biohazard symbol

After injecting Voxzogo

  • Check the injection site. If you see a small amount of blood at the injection site, gently press with a gauze pad for several seconds or apply a bandage.
  • Do not rub the injection site.
  • Watch for signs of low blood pressure, such as dizziness, tiredness, or feeling unwell. If you have these symptoms, call your doctor or healthcare professional, then lie down on your back and place pillows under your legs to keep them elevated.

How to dispose of Voxzogo

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

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

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

When the container you are using for sharps disposal is nearly full, follow local guidelines for proper disposal.

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