QALSODY 100 mg INJECTABLE SOLUTION

Introduction

Package Leaflet: Information for the Patient

Qalsody 100 mg solution for injection

tofersen

This medicine is subject to additional monitoring, which will allow for quick identification of new safety information. You can help by reporting any side effects you may get. The last section of section 4 will tell you how to report side effects.

Read all of this leaflet carefully before you are given this medicine because it contains important information for you.

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

Contents of the pack

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

1. What is Qalsody and what is it used for

Qalsody contains the active substance tofersen, which belongs to a group of medicines known as antisense oligonucleotides.

This medicine is used in adults to treat a type of amyotrophic lateral sclerosis(ALS) caused by mutations (changes) in a gene called SOD1.

The ALS caused by mutations in the SOD1 gene is a rare type of motor neuron disease that affects the nerve cells in the brain and spinal cord. The mutations in the SOD1 gene produce an accumulation of a toxic form of the SOD1 protein. This causes the destruction of motor neurons (the nerve cells responsible for sending instructions to the muscle), leading to muscle weakness and atrophy, including those used for breathing and swallowing.

Qalsody reduces the accumulation of the SOD1 protein. This helps to prevent the destruction of motor neurons and may slow down the loss of muscle strength.

2. What you need to know before you are given Qalsody

Qalsody must not be given

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

Talk to your doctor or nursebefore starting treatment if this applies to you.

Warnings and precautions

There is a risk that side effects may occur after the administration of Qalsody by the lumbar puncture procedure (see section 3). These may include headache, back pain, and infection.

A small number of cases of patients who have developed inflammation of the spinal cord (myelitis) or irritation or injury of the nerve roots (radiculitis) after the administration of Qalsody have been reported. You should be aware of the symptoms of these conditions while you are being treated with this medicine. See serious side effectsin section 4 of this leaflet.

A small number of cases of patients who have developed swelling of the optic nerve of the eye (papilledema) or an increase in pressure around the brain (increased intracranial pressure) have been reported in patients treated with Qalsody. See serious side effectsin section 4 of this leaflet.

Tests before treatment

You may have a urine test(to check your kidneys) and a blood test(to check that your blood is clotting properly) before starting treatment. This is because other medicines in the same group as Qalsody may affect the kidneys and the blood cells that help with clotting. You may not need to have these tests every time you are given Qalsody.

Children and adolescents

This medicine must not be given to children and adolescents under 18 years of age. The use of this medicine in patients under 18 years of age has not been studied.

Other medicines and Qalsody

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

Pregnancy and breastfeeding

If you are pregnant or breastfeeding, think you may be pregnant, or are planning to have a baby, ask your doctor for advice before you are given this medicine.

Pregnancy

Qalsody is not recommended during pregnancy, or in women of childbearing potential who are not using contraception.

Breastfeeding

Your doctor will help you decide whether to continue breastfeeding or start treatment with Qalsody. Your doctor will consider the potential benefits of treatment for you compared to the benefits of breastfeeding for your baby.

Driving and using machines

This medicine may affect your ability to drive or use machines. Do not drive or use machines if you notice a change in your vision with Qalsody.

Qalsody contains sodium

This medicine contains 52 mg of sodium (the main component of cooking/table salt) in each 15 ml. This is equivalent to 3% of the maximum recommended daily intake of sodium for an adult.

Qalsody contains potassium

This medicine contains less than 1 mmol (39 mg) of potassium per 15 ml dose; this is essentially “potassium-free”.

3. How Qalsody is given

The recommended dose is 100 mg of tofersen. The first three doses are given at intervals of 14 days between them on day 1, day 15, and day 29 of treatment. After that, Qalsody will be given every 28 days.

This medicine is given by injection into the spinal fluid (in the space surrounding the spinal cord) in the lower back through a lumbar puncture. This will be done by a doctor with experience in lumbar punctures.

How long Qalsody is used

Your doctor will tell you how long you should receive Qalsody. Do not stop treatment with Qalsody without talking to your doctor.

If you miss a dose of Qalsody

If you miss a dose of Qalsody, talk to your doctor so that it can be given as soon as possible.

If you have any other questions about the use of this medicine, ask your doctor, pharmacist, or nurse.

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 during or after the administration of Qalsody. These side effects may include headache, back pain, and infection.

Serious side effects

The most serious side effects seen in patients treated with Qalsody are inflammation of the spinal cord (myelitis) or irritation and injury of the nerve roots (radiculitis). Common symptoms may include:

• weakness
• numbness
• abnormal sensations (tingling)
• pain.

Swelling of the nerve that connects the eyes to the brain (papilledema) and increased pressure around the brain (increased intracranial pressure) have also been reported. Papilledema may be a result of increased intracranial pressure. Common symptoms may include:

• blurred vision
• double vision
• loss of vision
• headache.

Cases of inflammation of the tissue that covers the brain and spinal cord (aseptic or chemical meningitis) have been reported. This is not due to an infection. Common symptoms may include:

• headache
• fever
• stiff neck
• nausea
• vomiting.

Tell your doctor immediatelyif you get any of the symptoms mentioned above.

Other side effects

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

• pain (back pain, pain in the arms and legs)
• feeling tired
• muscle and joint pain
• fever
• increased protein or white blood cell count in the fluid that surrounds the brain and spinal cord.

Common(may affect up to 1 in 10 people)

• muscle stiffness
• nerve pain, including burning, stabbing, or tingling sensations.

Tell your doctorif you get these symptoms or any other new symptoms that worry you.

Reporting of side effects

If you get any side effects, talk to your doctor, pharmacist, 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 in the European Medicines Agency website. By reporting side effects, you can help provide more information on the safety of this medicine.

5. Storage of Qalsody

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 carton after EXP. The expiry date refers to the last day of the month shown.

Do not use this medicine if you notice particles in the solution or if the liquid in the vial is not clear and colorless.

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

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

The Qalsody vial can be stored at room temperature (below 30°C) for a maximum of 14 days.

Unopened Qalsody vials can be removed from and returned to the refrigerator if necessary. Unopened vials can be out of the carton for a maximum of 6 hours at room temperature for a maximum of 6 days.

6. Contents of the pack and other information

What Qalsody contains

• The active substance is tofersen.

• Each 15 ml vial contains 100 mg of tofersen.

• Each ml contains 6.7 mg of tofersen.

• The other ingredients are disodium phosphate, potassium chloride, calcium chloride dihydrate, magnesium chloride hexahydrate, sodium chloride, sodium dihydrogen phosphate dihydrate, water for injections.

Appearance and pack contents

Qalsody is a clear and colorless to slightly yellowish solution for injection.

Each pack of Qalsody contains one vial.

Marketing authorisation holder and manufacturer

Biogen Netherlands B.V.

Prins Mauritslaan 13

1171 LP Badhoevedorp

Netherlands

For further information about this medicine, contact the local representative of the marketing authorisation holder:

This medicine has been authorized under exceptional circumstances. This means that due to the rarity of the disease, it has not been possible to obtain complete information on this medicine. The European Medicines Agency will review any new information that may become available every year and this leaflet will be updated as necessary.

Date of last revision of this leaflet: 05/2024

Other sources of information

Detailed information on this medicine is available on the European Medicines Agency website.