Evrysdi ® ; 0,75 mg Oral Solution

What Evrysdi is

Evrysdi contains the active substance ‘risdiplam’, which is a selective SMN2 gene splicing modifier. Risdiplam can increase the amount of functional survival motor neuron (SMN) protein throughout the body.

What is Evrysdi used for

Evrysdi is used for the treatment of a genetic disease called ‘spinal muscular atrophy’ (SMA).

What spinal muscular atrophy is

SMA is caused by a shortage of a protein called ‘survival motor neuron’ (SMN) in the body. If you do not have enough SMN protein, you lose motor neurons. This leads to muscle weakness and wasting. This may affect everyday movements such as head and neck control, sitting, crawling and walking. The muscles used for breathing and swallowing may also be affected.

How Evrysdi works

Evrysdi works by helping the body produce more functional SMN protein. This means less motor nerves are lost, which may improve muscle strength and function in people across a broad range of ages and SMA types. In infants with SMA, Evrysdi may improve survival, preserve swallowing, and reduce the need for hospitalisation and ventilator supported breathing. In children (toddlers to adolescents) and adults, Evrysdi may maintain or improve motor function over time.

Do not take Evrysdi:

• If you are allergic to risdiplam or any of the other ingredients of this medicine.
• If you or your partner are pregnant or are planning to become pregnant. If you are not sure, talk to your medical practitioner or pharmacist before you take Evrysdi.

Warnings and precautions

Talk to your medical practitioner, nurse or pharmacist before taking Evrysdi. Treatment with Evrysdi may harm your unborn baby or may affect male fertility. See “Pregnancy, contraception, breast-feeding and male-fertility” in this section for more information.

Other medicines and Evrysdi

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

Pregnancy, contraception, breastfeeding and male fertility

Pregnancy

• If you are pregnant, think you may be pregnant or are planning to have a baby, ask your medical practitioner or pharmacist for advice before taking this medicine. This is because taking this medicine while you are pregnant could harm your unborn baby.
• Before you start treatment with Evrysdi, your medical practitioner may do a pregnancy test. This is because Evrysdi may harm your unborn baby. Your doctor will consider the benefit of you taking Evrysdi against the risk to your baby.
• If you do become pregnant during your treatment with Evrysdi, tell your medical practitioner straight away. You and your medical practitioner will decide what is best for you and your unborn baby.

Contraception

For women

• Do not become pregnant during your treatment with Evrysdi and for one month after you stop taking Evrysdi. Talk to your medical practitioner about reliable methods of birth control that should be used during treatment and for one month after you stop treatment.

For men

• If your female partner is of childbearing potential, you need to avoid pregnancy. Use reliable methods of control (eg. Condoms): during your treatment with Evrysdi and for 4 months after you stop taking Evrysdi. Talk to your healthcare provider about reliable methods of birth control that should be used.

Breastfeeding

Do not breastfeed while taking this medicine. This is because Evrysdi may pass into breast milk and may therefore harm your baby. Discuss with your doctor if you should stop breastfeeding while taking Evrysdi.

Male fertility

Evrysdi may affect male fertility. For your family planning, ask your medical practitioner for advice. Do not donate sperm during your treatment and for 4 months after you stop taking Evrysdi.

Driving and using machines

Evrysdi is unlikely to affect your ability to drive and use machines. It is not always possible to predict to what extent Evrysdi may interfere with the daily activities of a patient. Patients should ensure that they do not engage in the above activities until they are aware of the measure to which Evrysdi affects them.

Evrysdi contains sodium

Evrysdi contains a small amount of sodium (salt) – there is less than 1 mmol (23 mg) sodium even at the highest daily dose of 5 mg (6,6 mL of 0,75 mg/mL oral solution). This means it is essentially ‘sodium-free’ and can be used by people on a sodium-restricted diet.

Evrysdi contains 0,375 mg of sodium benzoate per mL. Sodium benzoate may increase jaundice (yellowing of the skin and eyes) in newborn babies (up to 4 weeks old).

Evrysdi contains isomalt

Evrysdi contains 2,97 mg of isomalt per mL. If you have been told by your doctor that you or your child have an intolerance to some sugars, contact your doctor before taking this medicinal product.

Always take this medicine exactly as your doctor or pharmacist has told you. Check with your doctor or pharmacist if you are not sure.

You must also carefully read and follow the enclosed “Instructions for use” Booklet on how to take or give Evrysdi.

When and how to take Evrysdi

• Evrysdi is a liquid, and is referred to as a ‘solution’ or ‘medicine’ in this leaflet.
• Take Evrysdi once daily at approximately the same time each day. This will help you remember when to take your medicine.
• You can take Evrysdi with or without food.

How much Evrysdi to take

• Adolescents and adults: The daily dose of Evrysdi is 5 mg (6,6 mL of the 0,75 mg/mL oral solution).
• Infants and children: Your doctor will work out daily dose of Evrysdi based on your child’s age and weight. You must take your daily dose as instructed by your doctor. Do not change the dose without speaking to your doctor.

If you do not swallow the full dose, or vomit after taking a dose of Evrysdi, do not take an extra dose. Instead, just take the next dose at the usual time the next day.

Read the ‘Instructions for use’ Booklet

You can find a separate “Instructions for use” Booklet in the pack. This booklet gives detailed instructions on how to prepare your dose volume with the re-usable oral syringe provided, and take the medicine either:

• by mouth, or
• through a gastrostomy tube, or
• through a nasogastric tube.

How long to take Evrysdi for

Your doctor will tell you how long to take Evrysdi for. Do not stop treatment with Evrysdi unless your doctor tells you to.

If you take more Evrysdi than you should

If you take more Evrysdi than you should, talk to a doctor or go to hospital straight away. Take the medicine pack and this leaflet with you.

If you forget to take Evrysdi

• If it is within 6 hours of when you normally take Evrysdi, take the missed dose as soon as you remember.
• If it is over 6 hours from when you normally take Evrysdi, skip the missed dose and then take your next dose at the usual time. Do not take a double dose to make up for a forgotten dose.

Evrysdi can have side effects. Not all side effects reported for Evrysdi are included in this leaflet. Should your general health worsen or if you experience any untoward effects while taking Evrysdi, please consult your doctor, pharmacist or other healthcare professional for advice.

Very common: may affect more than 1 in 10 people

• diarrhoea
• rash
• headache
• fever

Common: may affect up to 1 in 10 people

• nausea
• mouth sores
• bladder infection
• joint pain

Unknown (frequency cannot be estimated from the available data):

• inflammation of small blood vessel mainly affecting the skin (cutaneous vasculitis)

Reporting of side effects

If you get any side effects, talk to your doctor or pharmacist or nurse. You can also report side effects to SAHPRA via the Med Safety APP (Medsafety X SAHPRA) and eReporting platform (who-umc.org) found on SAHPRA website. By reporting side effects, you can help provide more information on the safety of Evrysdi.

• Keep this medicine and the re-usable oral syringes out of the sight and reach of children.
• Do not store the powder for oral solution above 25 °C.
• Store the oral solution in a refrigerator (between 2 ˚ C and 8 ˚ C). Do not freeze.
• If necessary, you or your caregiver may store the oral solution at room temperature (below 40 °C) for no more than a total combined time of 5 days.
• Keep the medicine in the original bottle.
• Keep the oral solution in the original amber bottle and keep bottle in an upright position with the cap tightly closed.
• Once transferred from the bottle to the oral syringe, use Evrysdi straight away.
• Do not store the Evrysdi solution in the syringe.
• Wash the syringes per instructions after use. Do not throw the syringes away.
• The oral solution is stable for 64 days after constitution by your healthcare provider when stored in the refrigerator at 2 ˚ C and 8 ˚ C. The pharmacist will write the date of expiration on the bottle label and on the original carton after “Discard after” date written on the bottle label or discard the medicine if the bottle has been stored at room temperature (below 40 °C) for more than a total combined time of 5 days. Discard the medicine if the bottle has been stored at above 40 °C.
• Do not use the solution past this “Discard after” date.
• Do not throw away any medicines via wastewater or household waste. Ask your pharmacist how to throw away medicines you no longer use. These measures will help protect the environment.