QALSODY

Package leaflet: Information for the patient

Qalsody 100 mg solution for injection

tofersen
This medicinal product is subject to additional monitoring. This will allow for the rapid identification of new safety information. You can help by reporting any side effects you experience while taking this medicine. See the end of section 4 for information on how to report side effects.
Please read this leaflet carefully before receiving this medicine as it contains important information for you.

• Keep this leaflet. You may need to read it again.
• If you have any questions, ask your doctor, pharmacist, or nurse.
• If you experience any side effects, including those not listed in this leaflet, tell your doctor, pharmacist, or nurse. See section 4.

Contents of this leaflet

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

1. What Qalsody is and what it is 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 for the treatment of a type of amyotrophic lateral sclerosis (ALS)
caused by mutations (changes) in a gene called SOD1.
ALS caused by mutations in the SOD1 gene is a rare type of motor neuron disease that affects nerve
cells in the brain and spinal cord.
Mutations in the SOD1 gene cause the accumulation of a toxic form of the SOD1 protein. This leads
to the destruction of motor neurons (nerve cells responsible for sending signals to muscles), resulting
in muscle weakness and loss of muscle mass, including in muscles used for breathing and
swallowing.
Qalsody works by reducing the accumulation of the SOD1 protein. This helps prevent the
destruction of motor neurons and may slow down the loss of muscle strength.

2. What you should know before receiving Qalsody

You must not be given Qalsody

• if you are allergic to tofersen or to any of the other ingredients of this medicine (listed in section 6). Consult your doctor or nurse before starting treatment if this applies to you.

Warnings and precautions
There is a risk of adverse reactions following administration of Qalsody by lumbar puncture
(see section 3). These may include headache, back pain, and infection.
In a small number of patients, inflammation of the spinal cord ( myelitis ) or irritation or damage to nerve roots ( radiculitis ) has been reported after administration of Qalsody.
You need to be aware of the symptoms during treatment with this medicine.
See Serious adverse reactions in section 4 of this package leaflet.
In a small number of patients treated with Qalsody, swelling of the optic nerve in the eye ( papilledema ) and/or increased pressure around the brain (increased intracranial pressure) has been reported. See Serious adverse reactions in section 4 of this package leaflet.

Tests before treatment
You may need to undergo a urine test (to check kidney function) and a blood test (to check that blood clotting is functioning properly) before starting treatment. This is because other medicines in the same class as Qalsody may affect the kidneys and blood cells involved in clotting. These tests may not be required every time you receive 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
Inform your doctor if you are taking, have recently taken, or might take any other medicines.

Pregnancy and breastfeeding
If you are pregnant, think you might be pregnant, planning to become pregnant, or breastfeeding, consult your doctor before receiving this medicine.

Pregnancy
The use of Qalsody is not recommended during pregnancy or in women of childbearing potential who are not using contraceptive measures.

Breastfeeding
Your doctor will help you decide whether to continue breastfeeding or start treatment with Qalsody. Your doctor will evaluate the potential benefit of therapy for you and the benefit of breastfeeding for the infant.

Driving and using machines
This medicine may affect your ability to drive or use machinery.
Do not drive or operate machinery if you experience vision disturbances while taking Qalsody.

Qalsody contains sodium
This medicine contains 52 mg of sodium (a main component of table salt) per 15 mL.
This corresponds to 3% of the maximum daily recommended dietary intake for an adult.

Qalsody contains potassium
This medicine contains less than 1 mmol (39 mg) of potassium per 15 mL, i.e. essentially ‘potassium-free’.

3. How Qalsody is administered

The recommended dose is 100 mg of tofersen. The first three doses will be administered at
14-day intervals on day 1, day 15, and day 29 of treatment. After that, Qalsody will be
administered every 28 days.
This medicine is administered by intrathecal injection (into the fluid surrounding the spinal cord) in the lower back via lumbar puncture. The procedure is performed by a physician experienced in lumbar punctures.
How long Qalsody is used
Your doctor will discuss with you how long treatment with Qalsody is needed. Do not stop treatment
with Qalsody without consulting your doctor.
If you miss an injection of Qalsody
If you miss a dose of Qalsody, contact your doctor so that the missed dose can be administered
as soon as possible.
If you have any doubts about the use of this medicine, consult 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 lumbar puncture may occur during administration of Qalsody or afterwards. Side effects may include headache, back pain, and infection.

Serious side effects
The most serious side effects observed in patients treated with Qalsody have been inflammation of the spinal cord ( myelitis ) or irritation and damage to the nerve roots ( radiculitis ). Common symptoms may include:

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

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

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

Inflammation of the membrane surrounding the brain and spinal cord ( aseptic or chemical meningitis ), not caused by infection, has been reported. Common symptoms may include:

• headache
• fever
• neck stiffness
• nausea
• vomiting.

Contact your doctor immediately if any of the symptoms listed above occur.

Other side effects

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

• pain (back pain, pain in arms or legs)
• feeling of fatigue
• muscle and joint pain
• fever
• increased protein and/or number of white blood cells in the fluid surrounding the brain and spinal cord.

Common (may affect up to 1 in 10 people)

• muscle weakness
• nerve pain, including burning sensations, shooting pain, and tingling.

Inform your doctor if you experience any of these symptoms or any new symptoms that concern you.

Reporting of side effects
If you experience any side effect, including those not listed in this leaflet, talk to your doctor, pharmacist or nurse. You can also report side effects directly via the national reporting system listed in Annex V. By reporting side effects, you can help provide more information on the safety of this medicine.

5. How to store 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 that month.
Do not use this medicine if you see particles in the solution or if the liquid in the vial is not
clear and colourless.
Store in a refrigerator (2 °C – 8 °C). Do not freeze.
Keep the vial in the outer packaging to protect the medicine from light.
The Qalsody vial in its original cardboard packaging can be stored for up to
14 days at room temperature (store below 30 °C).
Unopened Qalsody vials may be removed from the refrigerator and returned to the refrigerator, if
necessary. Unopened vials may be kept outside the original cardboard packaging for
no more than 6 hours per day at room temperature, for a maximum of 6 days.

6. Package contents 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 components are disodium phosphate, potassium chloride, calcium chloride dihydrate, magnesium chloride hexahydrate, sodium chloride, sodium dihydrogen phosphate dihydrate, water for injections.

Description of the appearance of Qalsody and contents of the pack
Qalsody is a clear, colourless to slightly yellow injectable solution.
Each Qalsody pack contains one vial.

Marketing Authorisation Holder and Manufacturer
Biogen Netherlands B.V.
Prins Mauritslaan 13
1171 LP Badhoevedorp
The Netherlands

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

België/Belgique/Belgien Lietuva
Biogen Belgium NV/SA Biogen Lithuania UAB
Tél/Tel: +32 2 2191218 Tel: + 370 5 259 6176

България Luxembourg/Luxemburg
ТП БЪЛГАРИЯ Biogen Belgium NV/SA
Teл.: +359 2 962 12 00 Tél/Tel: +32 2 2191218

Česká republika Magyarország
Biogen (Czech Republic) s.r.o. Biogen Hungary Kft.
Tel: +420 255 706 200 Tel.: +36 1 899 9883

Danmark Malta
Biogen (Denmark) A/S Pharma. MT Ltd.
Tlf: + 45 77 41 57 57 Tel: + 356 21337008

Deutschland Nederland
Biogen GmbH Biogen Netherlands B.V.
Tel: + 49 (0) 89 99 6170 Tel: + 31 20 542 2000

Eesti Norge
Biogen Estonia OÜ Biogen Norway AS
Tel: + 372 618 9551 Tlf: + 47 23 40 01 00

Ελλάδα Österreich
Genesis Pharma SA Biogen Austria GmbH
Τηλ: + 30 210 8771500 Tel: + 43 1 484 46 13

España Polska
Biogen Spain, S.L. Biogen Poland Sp. z o.o.
Tel: + 34 91 310 7110 Tel.: + 48 22 351 51 00

France Portugal
Biogen France SAS Biogen Portugal Sociedade Farmacêutica,
Tél: + 33 (0)1 41 37 95 95 Unipessoal, Lda.
Tel: + 351 21 318 8450

Hrvatska România
Biogen Pharma d.o.o. Ewopharma România SRL
Tel: + 385 (0) 1 775 73 22 Tel: + 40 (0)21 260 13 44

Ireland Slovenija
Biogen Idec (Ireland) Ltd. Biogen Pharma d.o.o.
Tel: + 353 (0)1 463 7799 Tel: + 386 1 511 02 90

Ísland Slovenská republika
Icepharma hf Biogen Slovakia s.r.o.
Sími: + 354 540 8000 Tel: + 421 2 323 340 08

Italia Suomi/Finland
Biogen Italia s.r.l. Biogen Finland Oy
Tel: + 39 02 5849901 Puh/Tel: + 358 207 401 200

Κύπρος Sverige
Genesis Pharma Cyprus Ltd Biogen Sweden AB
Τηλ: + 357 22765715 Tel: + 46 8 594 113 60

Latvija
Biogen Latvia SIA
Tel: + 371 68 688 158

This medicinal product has been authorised under “exceptional circumstances”. This means that, due to the rarity of the disease, it has not been possible to obtain complete information about this medicinal product.
The European Medicines Agency will review any new information on this medicinal product annually, and this package leaflet will be updated if necessary.

Further sources of information
More detailed information on this medicinal product is available on the website of the European Medicines Agency: https://www.ema.europa.eu/