Zolgensma – Onasemnogen-abeparvovek uses, dose and side effects


2 × 10 13  vector genome/ml infusion fluid ,solution

What Zolgensma is and what it is used for

What Zolgensma is

Zolgensma is a type of medicine called gene therapy. It contains the active substance caseinogen-abeparvovek, 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 is caused by a lack of an abnormal version of a gene needed to make a necessary protein called survival motor neuron (SMN). The absence of SMN protein means that nerves that control muscles (motor neurons) die. This leads to the muscles becoming weak and atrophied and ultimately to loss of mobility.

This medicine works by providing a fully functional copy of the SMN gene which then helps the body produce enough SMN protein. The genes are delivered into the cells where they are needed using a modified virus that does not cause disease in humans.

What you need to know before your child receives Zolgensma

Do NOT use Zolgensma

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

Warnings and precautions

Your child’s doctor will test for antibodies before treatment to determine if this medicine is suitable for your child.

Liver problems

Talk to your child’s doctor or nurse before giving this medicine if your child has had any liver problems. This medicine can lead to an increase in the enzymes ( proteins found in the body) produced in the liver or damage to the liver. Possible signs to look out for after your child has been given this medicine include vomiting, jaundice (yellowing of the skin or the whites of the eyes), or decreased 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 at least 3 months after treatment to check for increases in liver enzymes.


An infection (eg cold, flu, or bronchiolitis) before or after Zolgensma treatment can lead to more serious complications. You must pay attention to signs of an infection such as cough, wheezing, sneezing, runny nose, sore throat, or fever. Tell your child’s doctor immediately if you notice that your child develops any of these symptoms.

Regular blood tests

This medicine can reduce the number of blood platelets ( thrombocytopenia ). You need to be aware of any signs of low platelet count after your child has received Zolgensma such as abnormal bruising or bleeding (see section 4 for more information). Most of the cases of low platelet counts that have been reported occurred within two weeks of the child receiving Zolgensma.

Zolgensma can increase the levels of a heart protein called “troponin I” and maybe a sign of damage to the heart. You need to watch out for any signs of heart problems after your child has been given this medicine, such as pale gray or blue skin color, difficulty breathing, and swelling of the arms and legs or stomach (see section 4 for more information).

Before starting treatment with Zolgensma, your child will have a blood test to check the number of blood cells (including red blood cells and platelets ) and the troponin I level in the body. They will also have blood tests to check the creatinine level, which is a measure of how well the kidneys are working. Sometime after treatment, your child will also have regular blood tests to check 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 coagulation ( platelets ). These blood clots can affect your baby’s kidneys. Your child’s doctor may want to check your child’s blood ( platelet count ) and blood pressure. Possible signs to look out for after your child has received Zolgensma include bruising easily, convulsions (seizures), or a decrease in the amount of urine (see section 4 for more information). Contact a doctor immediately if your child develops any of these signs.

Blood, organ, tissue, and cell donation

After your child has been treated with Zolgensma, he/she will not be able to donate blood, organs, tissue, 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 be taking other medicines.


For about 2 months or longer, your child will also receive a corticosteroid medicine eg. prednisolone (see also section 3) as part of Zolgensma treatment. The corticosteroid medicine helps treat increases in liver enzymes that your child may develop after receiving Zolgensma.


Because corticosteroids can affect the body’s immune system (defense system)

your child’s doctor may decide to delay certain vaccinations

during the time the child receives corticosteroid treatment. Talk to your child’s doctor or nurse if you have any questions.

Zolgensma contains sodium

This medicinal product contains 4.6 mg of sodium per ml corresponding to 0.23% of the WHO maximum recommended daily intake (2 grams of sodium for adults). 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 save living motor neurons but does not save dead motor neurons. Children with less severe symptoms of SMA (such as lack of reflexes or decreased muscle tone ) may have enough viable motor neurons to benefit significantly from treatment with Zolgensma. Zolgensma may not work as well in children with severe muscle weakness or paralysis, breathing problems or in children who cannot swallow, or children who have extensive malformations (eg heart defects), including patients with SMA type 0, as it may mean limited to improvement after treatment with Zolgensma. Your child’s doctor will decide whether your child should receive this medicine.

Hygiene care

The active substance in Zolgensma may be temporarily excreted via bodily fluids and feces from your child; this is called “excretion”. Parents and caregivers must observe good hand hygiene for up to 1 month after the child has received Zolgensma. Use protective gloves when in direct contact with the child’s body fluids or feces and wash your hands thoroughly afterward with soap and running warm water or an alcohol-based hand sanitizer. Double bags should be used to dispose of soiled diapers and other waste. Disposable diapers can still be thrown into 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 to use Zolgensma

Zolgensma is given by a doctor or nurse trained in treating your child’s condition.

The doctor will calculate the amount of Zolgensma your child should receive according to the child’s weight. Zolgensma is given intravenously (into a vein) as a single infusion (drip) over about 1 hour.

Zolgensma is only given ONCE to your child.

Your child will also receive prednisolone (or another corticosteroid ) by mouth starting 24 hours before he/she receives Zolgensma. The dose of a corticosteroid also depends on the child’s weight. Your child’s doctor will work out the total dose to be given.

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

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

Possible side effects

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

Contact a doctor immediately if your child develops any of the following serious side effects:

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

  • bruising or bleeding that lasts longer than usual if the child has been ill – these may be signs of low platelet counts
  • pale gray or blue skin color, breathing problems (eg rapid breathing, shortness of breath), swelling of the arms and legs or stomach – these may be signs of possible problems with the heart.

Has been reported (occurring in an unknown number of users):

  • vomiting, jaundice (yellowing of the skin or the whites of the eyes), or decreased alertness – these may be signs of liver damage
  • bruising easily, convulsions (seizures), or a decrease in the amount of urine – these may be signs of thrombotic microangiopathy.

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

Very common (may affect more than 1 in 10 users):

  • increases in liver enzymes seen in blood tests.

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

  • vomiting
  • fever.

How to store Zolgensma

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

The following information is intended for healthcare professionals who will prepare and administer the medicine.

Do not use before the expiry date which is stated on the vial and carton and the label after EXP. The expiration date is the last day of the specified month.

Vials must be transported deep-frozen (at or below −60 °C).

Upon receipt, vials should be immediately refrigerated at 2°C to 8°C and stored in the original carton. Treatment with Zolgensma must be started within 14 days of receiving the vials.

This medicine contains genetically modified organisms. Unused medicine or waste must be disposed of according to local guidelines for handling biological waste. As this medicine will be given by a doctor, the doctor is responsible for the proper disposal of the product. These measures will help to protect the environment.

Contents of the packaging and other information

Contents declaration

  • The active substance is onasemnogen-abeparvovek. Each vial contains caseinogen-apeparvovek at a nominal concentration of 2 × 10 13  vector genomes/ml.
  • Other ingredients are tromethamine, magnesium chloride, sodium chloride, poloxamer 188 hydrochloric acid (for pH adjustment), and water for injections.

Appearance and package sizes of the medicine

Zolgensma is a clear to slightly opaque, colorless to slightly whitish solution for infusion.

Zolgensma is supplied in vials containing a nominal fill volume of either 5.5 ml or 8.3 ml. Each vial is for single use only.

Each carton contains between 2 and 14 vials.

Marketing authorization holder and manufacturer

Novartis Europharm Limited

Vista Building

Elm Park, Merrion Road

Dublin 4



Almac Pharma Services Limited

Finnabair Industrial Estate

Dundalk, Co. Louth

A91 P9KD


Contact the representative of the Marketing Authorization Holder if you would like to know more about this medicine:

België/Belgique/BelgienNovartis Pharma NVTél/Tel: +32 2 246 16 11LithuaniaSIA Novartis Baltics Lietuvos filialasPhone: +370 5 269 16 50
BulgariaNovartis Bulgaria EOODPhone: +359 2 489 98 28Luxembourg/LuxembourgNovartis Pharma NVTél/Tel: +32 2 246 16 11
Czech RepublicNovartis s.r.oPhone: +420 225 775 111MagyarországNovartis Hungária Kft.Tel.: +36 1 457 65 00
DenmarkNovartis Healthcare A/SPhone: +45 39 16 84 00MaltaNovartis Pharma Services Inc.Phone: +356 2122 2872
DeutschlandNovartis Pharma GmbHPhone: +49 911 273 0The NetherlandsNovartis Pharma BVPhone: +31 88 04 52 111
EstoniaSIA Novartis Baltics Eesti branch phone: +372 66 30 810NorwayNovartis Norway iPhone: +47 23 05 20 00
GreeceNovartis (Greece) AEBEPhone: +30 210 281 17 12AustriaNovartis Pharma GmbHPhone: +43 1 86 6570
SpainNovartis Farmacéutica, SAPhone: +34 93 306 42 00PolishNovartis Poland Sp. z o. iPhone: +48 22 375 4888
FranceNovartis Pharma SASTel: +33 1 55 47 66 00PortugalNovartis Farma – Produtos Farmacêuticos, SAPhone: +351 21 000 8600
HrvatskaNovartis Hrvatska dooTel. +385 1 6274 220RomaniaNovartis Pharma Services Romania SRLPhone: +40 21 31299 01
IrelandNovartis Ireland LimitedPhone: +353 1 260 12 55SlovenijaNovartis Pharma Services Inc.Phone: +386 1 300 75 50
IcelandVistor hf.Phone: +354 535 7000Slovenian RepublicNovartis Slovakia s.r.oPhone: +421 2 5542 5439
ItalyNovartis Pharma SpAPhone: +39 02 96 54 1Finland/FinlandNovartis Finland OyTel: +358 (0)10 6133 200
CyprusNovartis Pharma Services Inc.Phone: +357 22 690 690SwedenNovartis Sweden ABPhone: +46 8 732 32 00
LatviaSIA Novartis BalticsPhone: +371 67 887 070United Kingdom (Northern Ireland)Novartis Ireland LimitedPhone: +44 1276 698370

Leave a Reply