Spinraza 12 mg solution for injection

Package Leaflet: Information for the user

Introduction

Package leaflet: information for the user

Spinraza 12 mg solution for injection

nusinersen

Read the entire leaflet carefully before you or your child is given this medicine, because it contains important information for you.

• Keep this leaflet. You may need to read it again.
• If you have any questions, ask your doctor or nurse.
• If you or your child experience any side effects, talk to your doctor or nurse, even if it is not a side effect listed in this leaflet. See section 4.

Contents of the leaflet

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

1. What Spinraza is and what it is used for

Spinraza contains the active substance nusinersen, which belongs to a group of medicines called antisense oligonucleotides. Spinraza is used to treat a genetic disease called spinal muscular atrophy (SMA).

The cause of spinal muscular atrophy is a deficiency of a protein called survival motor neuron (SMN) in the body. This deficiency leads to the loss of neurons in the spinal cord, resulting in weakening of the muscles of the shoulders, hips, thighs, and upper back. It may also weaken the muscles used for breathing and swallowing.

Spinraza helps the body produce more of the SMN protein that is missing in people with SMA. This reduces the loss of neurons and may therefore improve muscle strength.

2. What you need to know before you or your child are given Spinraza

Do not receive Spinraza

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

If in doubt, consult your doctor or nurse before you or your child receive Spinraza.

Warnings and precautions

There is a risk of experiencing adverse effects following administration of Spinraza via a lumbar puncture procedure (see section 3). These adverse effects may include headaches, vomiting, and back pain. Difficulties in administering the medicine using this method may also occur in very young patients and in patients with scoliosis (curvature and deviation of the spine).

Other medicines belonging to the same class as Spinraza have been shown to affect blood cells involved in clotting. Before you or your child receive Spinraza, your doctor may decide to perform a blood test to check that your blood or your child's blood clots properly. This test may not be necessary every time Spinraza is administered to you or your child.

Other medicines in the same class as Spinraza have been shown to affect the kidneys. Before administration of Spinraza, your doctor may decide to perform a urine test to check that your kidneys are functioning properly. This test may not be necessary every time Spinraza is administered to you or your child.

A small number of cases of patients developing hydrocephalus (accumulation of excess fluid around the brain) after administration of Spinraza have been reported. Some of these patients required placement of a device called a ventriculoperitoneal shunt to treat the hydrocephalus. If you notice any symptoms such as an increase in head size, decreased level of consciousness, nausea, persistent headaches, vomiting, or any other symptoms of concern, inform your doctor or your child’s doctor immediately to receive appropriate treatment. Currently, the risks and benefits of continuing treatment with Spinraza in patients with a ventriculoperitoneal shunt are unknown.

Please consult your doctor before you or your child are given Spinraza.

Other medicines and Spinraza

Inform your doctor if you or your child are taking, have recently taken, or might need to take any other medicines in the future.

Pregnancy and breastfeeding

If you are pregnant or breastfeeding, think you may be pregnant, or are planning to become pregnant, consult your doctor before receiving this medicine. It is preferable to avoid using Spinraza during pregnancy and breastfeeding.

Driving and use of machines

The effect of Spinraza on the ability to drive and use machines is none or negligible.

Spinraza contains a small amount of sodium

This medicine contains less than 1 mmol of sodium (23 mg) per 5 ml vial; therefore, it is essentially “sodium-free” and can be used by individuals on sodium-restricted diets.

Spinraza contains a small amount of potassium

This medicine contains less than 1 mmol of potassium (39 mg) per 5 ml vial, and is therefore essentially “potassium-free”.

3. How Spinraza is administered

Spinraza is administered at a dose of 12 mg:

• On the first day of treatment, day 0.
• Then around day 14, day 28, and day 63.
• Afterwards, once every 4 months.

Spinraza is administered by injection into the lower back. This injection, called a lumbar puncture, is performed by inserting a needle into the space surrounding the spinal cord. A physician experienced in lumbar punctures will administer the injections. You or your child may also be given a medication to help relax or to sleep during the procedure.

How long Spinraza should be used

Your doctor will advise you on how long you or your child should continue receiving Spinraza. Do not stop treatment with Spinraza unless instructed by your doctor.

If you or your child misses an injection

If you or your child misses a dose of Spinraza, speak with your doctor so that Spinraza can be administered as soon as possible.

If you have any questions about how Spinraza is administered, ask your doctor.

4. Possible adverse effects

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

Adverse effects related to lumbar puncture may occur while receiving Spinraza or after administration. Most of these adverse effects are reported within 72 hours following the procedure.

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

• Back pain
• Headache
• Vomiting
• Fever (high temperature)

Frequency not known (cannot be estimated from available data)

• Serious infection associated with lumbar puncture (e.g., meningitis)
• Hydrocephalus (accumulation of excess fluid around the brain)
• Non-infectious meningitis (inflammation of the membrane surrounding the spinal cord and brain, which may present as neck stiffness, headache, fever, nausea, and vomiting)
• Hypersensitivity (an allergic or allergic-type reaction that may include swelling of the face, lips, or tongue, skin rash, or itching)
• Arachnoiditis (inflammation of the membrane surrounding the brain and spinal cord), which may cause lower back pain, or pain, numbness, or weakness in the legs

Reporting of adverse effects

If you or your child experience any 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 listed in Appendix V. By reporting adverse effects, you can help provide more information on the safety of this medicine.

5. Storage of Spinraza

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

Do not use this medicine after the expiry date stated on the vial and 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 the vial in the outer packaging to protect it from light.

If refrigeration is not possible, Spinraza may be stored in the original carton, protected from light, at a temperature not exceeding 30°C for a maximum of 14 days.

Unopened Spinraza vials may be removed from and returned to the refrigerator as necessary. If removed from the original carton, the total time the medicine is kept outside the refrigerator must not exceed 30 hours, at a temperature not exceeding 25°C.

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

6. Contents of the pack and other information

Composition of Spinraza

• The active substance is nusinersen.

Spinraza 12 mg

• Each 5 ml vial contains sodium nusinersen equivalent to 12 mg of nusinersen.
• Each ml contains 2.4 mg of nusinersen.
• The other components are sodium dihydrogen phosphate dihydrate, sodium hydrogen phosphate, sodium chloride (see section 2, "Spinraza contains a small amount of sodium"), potassium chloride (see section 2, "Spinraza contains a small amount of potassium"), calcium chloride dihydrate, magnesium chloride hexahydrate, sodium hydroxide, hydrochloric acid, water for injections.

Nature of the product and contents of the pack

Spinraza is a clear, colourless injectable solution.

Each Spinraza carton contains one vial.

Each vial is for single use in one patient only. Do not dilute.

Marketing Authorisation Holder and Manufacturer

Biogen Netherlands B.V.

Prins Mauritslaan 13

1171 LP Badhoevedorp

The Netherlands

For more information about this medicinal product, please contact the local representative of the Marketing Authorisation Holder:

Date of the most recent review of this summary:

Other sources of information

Detailed information on this medicine is available on the European Medicines Agency website: https://www.ema.europa.eu.

This information is intended for healthcare professionals only:

1. The Spinraza vial should be inspected visually for particles before administration. If particles are observed and/or if the liquid in the vial is not clear and colourless, the vial must not be used.

2. An aseptic technique must be followed when preparing the Spinraza solution for intrathecal administration.

3. Before administration, the vial should be removed from the refrigerator and allowed to reach room temperature (25°C), without using external heat sources.

4. If the vial remains unopened and the solution has not been used, it should be returned to the refrigerator.

5. Immediately before administration, remove the plastic cap and insert the syringe needle through the center of the stopper to withdraw the required volume. Spinraza must not be diluted. External filters are not required.

6. Spinraza is administered as an intrathecal bolus injection over 1 to 3 minutes, using a spinal anaesthesia needle.

7. The injection must not be administered into areas of skin showing signs of infection or inflammation.

8. It is recommended to withdraw an amount of cerebrospinal fluid (CSF) equivalent to the volume of Spinraza to be injected prior to administering Spinraza.

9. Once the solution has been drawn into the syringe, if it is not used within 6 hours, it must be discarded.

10. Disposal of unused medicine contained in the vial and of all materials that have come into contact with it must be carried out in accordance with local regulations.