Medicines Zolgensma 2×1013 Vector Genomes-mL Solution for Infusion Instruction Manual

Medicines Zolgensma 2×1013 Vector Genomes-mL Solution for Infusion

This medicine is subject to additional monitoring. This will allow quick identification of new safety information. You can help by reporting any side effects your child may get. See the end of section 4 for how to report side effects.

Read all of this leaflet carefully before your child is given this medicine because it contains important information

• Keep this leaflet. You may need to read it again.
• If you have any further questions, ask your child’s doctor or nurse.
• If your child gets any side effects, talk to your child’s doctor or nurse. This includes any possible side effects not listed in this leaflet. See section 4.

What is in this leaflet

1. What Zolgensma is and what it is used for
2. What you need to know before your child is given Zolgensma
3. How Zolgensma is given
4. Possible side effects
5. How to store Zolgensma
6. Contents of the pack and other information

What Zolgensma is and what it is used for

What Zolgensma is
Zolgensma is a type of medicine called a ‘gene therapy’. It contains the active substance onasemnogene abeparvovec, which contains human genetic material.

What Zolgensma is used for
Zolgensma is used to treat ‘spinal muscular atrophy’ (SMA), a rare, serious inherited disease.

How Zolgensma works
SMA occurs when there is a missing or abnormal version of a gene needed to make an essential protein called the ‘Survival Motor Neuron’ (SMN) protein. Lack of SMN protein causes nerves that control muscles (motor neurons) to die. This results in muscles becoming weak and wasting away, with eventual loss of movement.
This medicine works by supplying a fully functioning copy of the SMN gene which then helps the body produce enough SMN protein. The gene is delivered into the cells where it is needed using a modified virus that does not cause disease in humans.

What you need to know before your child is given Zolgensma

Do NOT use Zolgensma

• if your child is allergic to onasemnogene abeparvovec or any of the other ingredients of this medicine (listed in section 6).

Warnings and precautions

Your child’s doctor will check for antibodies before treatment to help decide if this medicine is suitable for your child.

Liver problems
Talk to your child’s doctor or nurse before this medicine is given if your child has had any liver problems. This medicine can lead to an increase in enzymes (proteins found within the body) produced by the liver or injury to the liver. Possible signs you need to look out for after your child is given this medicine include vomiting, jaundice (yellowing of the skin or of the whites of the eyes), or reduced alertness (see section 4 for more information).

Your child will have a blood test to check how well the liver is working before starting treatment with Zolgensma. Your child will also have regular blood tests for at least 3 months after treatment to monitor for increases in liver enzymes.

Infection
An infection (e.g. cold, flu or bronchiolitis) before or after Zolgensma treatment may lead to more serious complications. You need to look out for signs of an infection such as coughing, wheezing, sneezing, runny nose, sore throat or fever. Tell your child’s doctor straightaway if you notice your child develops any of these symptoms.

Regular blood tests
This medicine can lower blood-platelet counts (thrombocytopenia). You need to look out for possible signs of a low blood-platelet count after your child is given Zolgensma such as abnormal bruising or bleeding (see section 4 for more information).

Zolgensma can raise levels of a heart protein called troponin-I that may indicate injury to the heart. You need to look out for possible signs of heart problems after your child is given this medicine, such as pale grey or blue skin colour, difficulty in breathing, swelling of the arms and legs or of the belly (see section 4 for more information).

Before starting treatment with Zolgensma, your child will have a blood test to check the amount of blood cells (including red blood cells and platelets), as well as troponin-I level in their body. They will also have a blood test to check their creatinine level, which is an indicator of how the kidneys are working. Your child will also have regular blood tests for a period of time after treatment to monitor for changes in platelets and troponin-I levels.

Abnormal clotting of blood in small blood vessels (thrombotic microangiopathy)
There have been reports of patients developing thrombotic microangiopathy approximately one week after Zolgensma treatment. Thrombotic microangiopathy is accompanied by a decrease in red blood cells and cells involved in clotting (platelets). These blood clots could affect your child’s kidneys. Your child’s doctor may want to check your child’s blood (platelet counts) and blood pressure. Possible signs you need to look out for after your child is given Zolgensma include bruising easily, seizures (fits) or decrease in urine output (see section 4 for more information). Seek urgent medical attention if your child develops any of these signs.

Blood, organ, tissue and cell donation
After your child has been treated with Zolgensma, they will not be able to donate blood, organs, tissues or cells. This is because Zolgensma is a gene therapy medicine.

Other medicines and Zolgensma

Tell your child’s doctor or nurse if your child is taking, has recently taken or might take any other medicines.

Prednisolone
Your child will also be given a corticosteroid medicine such as prednisolone for about 2 months or longer (see also section 3) as part of Zolgensma treatment. The corticosteroid medicine will help manage any increase in liver enzymes that your child could develop after being given Zolgensma.

Vaccinations
As corticosteroids can affect the body’s immune (defense) system, your child’s doctor may decide to delay giving some vaccinations while your child is receiving corticosteroid treatment. Talk to your child’s doctor or nurse if you have any questions.

Zolgensma contains sodium
This medicine contains 4.6 mg sodium per mL, equivalent to 0.23% of the WHO- recommended maximum daily intake of 2 g sodium for an adult. Each 5.5-mL vial contains 25.3 mg sodium, and each 8.3-mL vial contains 38.2 mg sodium.

Additional information for parents/caregivers

Advanced SMA
Zolgensma can rescue living motor neurons but does not rescue dead motor neurons. Children with less severe symptoms of SMA (such as absent reflexes or reduced muscle tone) may have sufficient living motor neurons to benefit significantly from Zolgensma treatment. Zolgensma may not work as well in children with severe muscle weakness or paralysis, breathing problems or who are not able to swallow, or in children who have significant malformations (such as heart defects), including patients with SMA Type 0, as there may be limited potential improvement after treatment with Zolgensma. Your child’s doctor will decide if your child should be given this medicine.

Hygiene care
The active substance in Zolgensma may temporarily be excreted through your child’s bodily waste; this is called ‘shedding’. Parents and caregivers should follow good hand hygiene for up to 1 month after their child is given Zolgensma. Wear protective gloves when coming into direct contact with your child’s bodily fluids or waste and wash hands thoroughly afterward with soap and warm running water, or an alcohol-based hand sanitizer. Double bags should be used to dispose of soiled nappies and other waste. Disposable nappies may still be disposed of in the household waste.

You should continue to follow these instructions for at least 1 month after your child’s treatment with Zolgensma. Talk to your child’s doctor or nurse if you have any questions.

How Zolgensma is given

Zolgensma will be given by a doctor or nurse trained in the management of your child’s condition. The doctor will work out the amount of Zolgensma your child will receive according to your child’s weight. Zolgensma is given intravenously (into a vein) by a single infusion (drip) over about 1 hour.

Zolgensma will be given to your child ONCE only.
Your child will also be given prednisolone (or another corticosteroid) by mouth, starting 24 hours before being given Zolgensma. The dose of corticosteroids will also depend on your child’s weight. Your child’s doctor will work out the total dose to give.

Your child will be given corticosteroid treatment daily for about 2 months after the dose of Zolgensma, or until your child’s liver enzymes decrease to an acceptable level. The doctor will slowly reduce the dose of corticosteroids until treatment can be fully stopped.

If you have any further questions ask your child’s doctor or nurse.

Possible side effects

Like all medicines, this medicine can have side effects, although not everybody gets them. Seek urgent medical attention if your child develops any of the following serious side effects: Common (may affect up to 1 in 10 people)

• bruising or bleeding for longer than usual if your child has been hurt – these may be signs of a low blood-platelet count;
• pale grey or blue skin colour, difficulty in breathing (e.g. rapid breathing, shortness of breath), swelling of the arms and legs or of the belly – these may be signs of possible problems with the heart.

Not known (frequency cannot be estimated from the available data)

• vomiting, jaundice (yellowing of the skin or of the whites of the eyes) or reduced alertness -these may be signs of injury to the liver.
• bruising easily, seizures (fits), decrease in urine output – these may be signs of thrombotic microangiopathy.

Talk to your child’s doctor or nurse if your child develops any other side effects. These can include:

Very common (may affect more than 1 in 10 people)

• increases in liver enzymes seen in blood tests.

Common (may affect up to 1 in 10 people):

• vomiting;
• fever.

Reporting of side effects
If your child gets any side effects, talk to your child’s doctor or nurse. This includes any possible side effects not listed in this leaflet. You can also report side effects directly via the Yellow Card Scheme at: www.mhra.gov.uk/yellowcard or search for MHRA Yellow Card in the Google Play or Apple App Store. By reporting side effects, you can help provide more information on the safety of this medicine.

How to store Zolgensma

Keep this medicine out of the sight and reach of children.
The following information is for healthcare professionals who will prepare and give the medicine. Do not use this medicine after the expiry date which is stated on the vial label and the carton after EXP. The expiry date refers to the last day of that month. Vials will be transported frozen (at or below -60ºC). Upon receipt, vials should be refrigerated at 2°C to 8°C immediately, and in the original carton. Zolgensma therapy should be initiated within 14 days of receipt of vials.

This medicine contains genetically-modified organisms. Unused medicine or waste material must be disposed of in compliance with the local guidelines on the handling of biological waste. As this medicine will be given by a doctor, the doctor is responsible for the correct disposal of the product. These measures will help protect the environment.

Contents of the pack and other information

What Zolgensma contains

• The active substance is onasemnogene abeparvovec. Each vial contains onasemnogene abeparvovec with a nominal concentration of 2 × 1013 vector genomes/mL.
• The other ingredients are tromethamine, magnesium chloride, sodium chloride, poloxamer 188, hydrochloric acid (for pH adjustment) and water for injections.

What Zolgensma looks like and contents of the pack
Zolgensma is a clear to slightly opaque, colourless to faint white solution for infusion. Zolgensma may be supplied in vials containing a nominal fill volume of either of 5.5 mL or 8.3 mL. Each vial is for single use only.

Each carton will contain between 2 to 14 vials.

Marketing Authorisation Holder

Novartis Pharmaceuticals UK Limited
2nd Floor, The WestWorks Building, White City Place 195 Wood Lane London W12 7FQ United Kingdom

Manufacturer
Almac Pharma Services Limited Finnabair Industrial Estate Dundalk, Co. Louth A91 P9KD Ireland

For any information about this medicine, please contact the Marketing Authorisation Holder.

This leaflet was last revised in 10/2022
This medicine has been given ‘conditional approval’. This means that there is more evidence to come about this medicine. The Health Authority will review new information on this medicine at least every year and this leaflet will be updated as necessary.

The following information is intended for healthcare professionals only:

Important: Please refer to the Summary of Product Characteristics (SmPC) before using.

Each vial is for single use only.

This medicinal product contains genetically-modified organisms. Local guidelines on the handling of biological waste should be followed.

Handling

• Zolgensma should be handled aseptically under sterile conditions.
• Personal protective equipment (including gloves, safety goggles, laboratory coat, and sleeves) should be worn while handling or administering Zolgensma. Personnel should not work with Zolgensma if the skin is cut or scratched.
• All spills of Zolgensma must be wiped with absorbent gauze pads and the spill area must be disinfected using a bleach solution followed by alcohol wipes. All clean-up materials must be double-bagged and disposed of in accordance with local guidelines on the handling of biological waste.
• All materials that may have come in contact with Zolgensma (e.g. vial, all materials used for injection, including sterile drapes and needles) must be disposed of in accordance with local guidelines on the handling of biological waste.

Accidental exposure
Accidental exposure to Zolgensma must be avoided.

In case of accidental exposure to the skin, the affected area must be thoroughly cleansed with soap and water for at least 15 minutes. In case of accidental exposure to eyes, the affected area must be thoroughly flushed with water for at least 15 minutes.

Storage
Vials will be transported frozen (at or below -60ºC). Upon receipt, vials should be refrigerated at 2°C to 8°C immediately and in the original carton. Zolgensma therapy should be initiated within 14 days of receipt of vials. The date of receipt should be marked on the original carton before the product is stored in the refrigerator.

Preparation

Vials should be thawed before use:

• For packs containing up to 9 vials – thaw for approximately 12 hours in the refrigerator (2ºC to 8ºC) or 4 hours at room temperature (20°C to 25°C).
• For packs containing up to 14 vials – thaw for approximately 16 hours in the refrigerator (2ºC to 8ºC) or 6 hours at room temperature (20°C to 25°C).

Do not use Zolgensma unless thawed. Once thawed, the medicinal product should not be re-frozen. After thawing, gently swirl Zolgensma. Do NOT shake. Do not use this medicine if you notice any particles or discoloration once the frozen product has thawed and prior to administration. After thawing, Zolgensma should be given as soon as possible.

Administration
Zolgensma should be given to patients ONCE only.

The dose of Zolgensma and an exact number of vials required for each patient is calculated according to the patient’s weight (see SmPC sections 4.2 and 6.5). To administer Zolgensma, draw the entire dose volume into the syringe. Once the dose volume is drawn into the syringe it must be administered within 8 hours. Remove any air in the syringe before administering to the patient via intravenous infusion through a venous catheter. Insertion of a secondary (‘back-up’) catheter is recommended in case of blockage in the primary catheter.

Zolgensma should be administered with the syringe pump as a single intravenous infusion with a slow infusion of approximately 60 minutes. It should be administered as an intravenous infusion only. It should not be administered as a rapid intravenous injection or bolus. Following completion of infusion, the line should be flushed with sodium chloride 9 mg/mL (0.9%) solution for injection.

Disposal
Any unused medicinal product or waste material should be disposed of in accordance with local guidelines on handling biological waste.

Temporary Zolgensma shedding may occur, primarily through bodily waste. Caregivers and patient’s families should be advised of the following instructions for the proper handling of patients’ bodily fluids and waste:

• Good hand hygiene (wearing protective gloves and washing hands thoroughly afterward with soap and warm running water, or an alcohol-based hand sanitizer) is required when coming into direct contact with the patient’s bodily fluids and waste for a minimum of 1 month after Zolgensma treatment.
• Disposable nappies should be sealed in double plastic bags and can be disposed of in the household waste.

Read User Manual Online (PDF format)

Read User Manual Online (PDF format)  >>

Download This Manual (PDF format)

Download this manual  >>