Spinraza - patient leaflet, side effects, dosage

1. What Spinraza is and what it is used for

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

Spinal muscular atrophy is caused by a shortage of a protein called survival motor neuron (SMN) in the body. This results in the loss of nerve cells in the spine, leading to weakness of the muscles in the shoulders, hips, thighs and upper back. It may also weaken the muscles used for breathing and swallowing.

Spinraza works by helping the body to produce more of the SMN protein that people with SMA are lacking. This reduces the loss of nerve cells and so may improve muscle strength.

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

Spinraza must not be given

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

If you are not sure, talk to your doctor or nurse before you or your child are given Spinraza.

Warnings and precautions

There is a risk of side effects occuring after Spinraza is given by a lumbar puncture procedure (see section 3). This can include headaches, vomiting and back pain. There may also be difficulties with giving a medicine by this method in very young patients and those with scoliosis (twisted and curved spine).

Other products that are in the same group of medicines as Spinraza have been shown to affect the cells in the blood which help clotting. Before you or your child are given Spinraza your doctor may decide to do a blood test to check that your or your child’s blood can clot properly. This may not be required every time you or your child are given Spinraza.

Other products that are in the same group of medicines as Spinraza have been shown to affect the kidneys. Before you or your child are given Spinraza your doctor may decide to do a urine test to check that your or your child’s kidneys are working normally. This may not be required every time you or your child are given Spinraza.

There have been a small number of reports of patients developing hydrocephalus (a build-up of too much fluid around the brain) after Spinraza is given. Some of these patients had needed to have a device called a ventriculo-peritoneal shunt implanted to treat the hydrocephalus. If you notice any symptoms of increase in head size, decreased consciousness, persistent nausea, vomiting or headache; or other symptoms that cause you concern, please inform your or your child’s doctor to seek necessary treatment. The benefits and risks of continuing Spinraza whilst having a „ventriculo-peritoneal shunt“ in place are not known at present.

Talk to your doctor before you or your child are given Spinraza.

Other medicines and Spinraza

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

Pregnancy and breast-feeding

If you are pregnant or breast-feeding, think you may be pregnant or are planning to have a baby, ask your doctor for advice before being given this medicine. It is preferable to avoid the use of Spinraza during pregnancy and breast-feeding.

Driving and using machines

Spinraza has no or negligible influence on the ability to drive and use machines.

Spinraza contains a small amount of sodium

This medicine contains less than 1 mmol sodium (23 mg) per 5 ml vial, that is to say essentially ‘sodium-free’ and can be used by people on a sodium-restricted diet.

Spinraza contains a small amount of potassium

This medicine contains potassium, less than 1 mmol (39 mg) per 5 ml vial, i.e. essentially ‘potassium-free’.

• 3. How Spinraza is given

The usual dose of Spinraza is 12 mg.

Spinraza is given;

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

Spinraza is given by injection into the lower back. This injection, called a lumbar puncture, is done by inserting a needle into the space around the spinal cord. This will be done by a doctor experienced in doing lumbar punctures. You or your child may also be given a medicine to make you relax or sleep during the procedure.

How long to use Spinraza

Your doctor will tell you how long you or your child need to receive Spinraza. Don’t stop treatment with Spinraza unless your doctor tells you to.

If you or your child misses an injection

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

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

4. Possible side effects

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

Side effects related to the lumbar puncture may occur while Spinraza is being given or afterwards. The majority of these side effects are reported within 72 hours of the procedure.

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

• Back pain
• Headache
• Vomiting

Additional side effects

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

• Serious infection related to lumbar puncture (e.g. meningitis)
• Hydrocephalus (a build-up of too much fluid around the brain)
• Meningitis not caused by an infection (inflammation of the membrane around the spinal cord and brain, which may present as neck stiffness, headache, fever, nausea and vomiting)
• Hypersensitivity (an allergic or allergic-like reaction that may include swelling of your face, lips or tongue, rash, or itching)

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 Spinraza

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 vial and carton after “EXP”. The expiry date refers to the last day of that month.

Store in a refrigerator (2°C to 8°C). Do not freeze.

Keep the vial in the outer carton in order to protect from light.

If no refrigeration is available, Spinraza may be stored in its original carton, protected from light at or below 30°C for up to 14 days.

Unopened vials of Spinraza can be removed from and returned to the refrigerator if necessary. If removed from the original carton, the total time out of refrigeration should not exceed 30 hours, at a temperature that does not exceed 25°C.

6. Contents of the pack and other information

What Spinraza contains

• – The active substance is nusinersen.

• – Each 5 ml vial contains nusinersen sodium equivalent to 12 mg nusinersen.

• – Each ml contains 2.4 mg of nusinersen.

• – The other ingredients are sodium dihydrogen phosphate dihydrate, disodium 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.

What Spinraza looks like and contents of the pack

Spinraza is a clear colourless solution for injection.

Each carton of Spinraza contains one vial.

Each vial is for single use.

Marketing Authorisation Holder

Biogen Netherlands B.V.

Prins Mauritslaan 13

1171 LP Badhoevedorp

The Netherlands

Manufacturer

FUJIFILM Diosynth Biotechnologies Denmark ApS

Biotek Allé 1

DK-3400 Hillerod

Denmark

Biogen Netherlands B.V.

Prins Mauritslaan 13

1171 LP Badhoevedorp

The Netherlands

Eesti Biogen Estonia OÜ Tel: + 372 618 9551	Norge Biogen Norway AS Tlf: +47 23 40 01 00
EXXáSa Genesis Pharma SA Tql: +30 210 8771500	Österreich Biogen Austria GmbH Tel: +43 1 484 46 13
España Biogen Spain SL Tel: +34 91 310 7110	Polska Biogen Poland Sp. z o.o. Tel.: +48 22 351 51 00
France Biogen France SAS Tél: +33 (0)1 41 37 95 95	Portugal Biogen Portugal Tel.: +351 21 318 8450
Hrvatska Biogen Pharma d.o.o. Tel: +385 (0) 1 775 73 22	Romania Ewopharma AG Representative Office Tel: + 40 377 881 045
Ireland Biogen Idec (Ireland) Ltd. Tel: +353 (0)1 463 7799	Slovenija Biogen Pharma d.o.o. Tel.: +386 1 511 02 90
Ísland Icepharma hf Sími: +354 540 8000	Slovenská republika Biogen Slovakia s.r.o. Tel.: +421 2 323 340 08
Italia Biogen Italia s.r.l. Tel: +39 02 584 9901	Suomi/Finland Biogen Finland Oy Puh/Tel: +358 207 401 200
Kúnpog Genesis Pharma Cyprus Ltd Tql: +357 22765715	Sverige Biogen Sweden AB Tel: +46 8 594 113 60
Latvija Biogen Latvia SIA Tel: + 371 68 688 158	United Kingdom (Northern Ireland) Biogen Idec (Ireland) Limited Tel: +44 (0) 1628 50 1000

This leaflet was last revised in

Other sources of information

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

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The following information is intended for healthcare professionals only:

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

• 2. Aseptic technique should be used when preparing Spinraza solution for intrathecal administration.

• 3. The vial should be taken out of the refrigerator and allowed to warm to room temperature (25°C) without using external heat sources, prior to administration.

• 4. If the vial remains unopened and the solution is not used, it should be returned back to the refrigerator.

• 5. Just prior to administration, remove the plastic cap and insert the syringe needle into the vial through the center of the over-seal to remove the appropriate volume. Spinraza must not be diluted. The use of external filters is 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 in areas of the skin where there are signs of infection or inflammation.

• 8. It is recommended that the volume of CSF, equivalent to the volume of Spinraza to be injected, is removed prior to administration of Spinraza.

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

• 10. Any unused product or waste material must be disposed of in accordance with local requirements.