Ezmekly 2 mg hard capsules – patient leaflet, side effects, dosage

1. What Ezmekly is and what it is used for

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

Ezmekly contains the active substance mirdametinib, and is a mitogen-activated protein kinase (MEK) inhibitor.

Ezmekly is used to treat plexiform neurofibromas in adults, adolescents, and children 2 years of age and older with a genetic condition called neurofibromatosis type 1 (NF1).

People with NF1 have a mutation (change) in the gene that encodes for the protein neurofibromin. This leads to a loss of this protein, which allows tumours to grow on the nerves in the body. Ezmekly works by blocking certain proteins involved in the growth of these tumour cells, which is expected to shrink them.

If you have any questions about how Ezmekly works or why this medicine has been prescribed for you, ask your doctor.

2. What you (or your child) need to know before you take Ezmekly

Do not take Ezmekly

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

Warnings and precautions

Talk to your doctor, pharmacist or nurse before taking Ezmekly, or if the following happen to you (or your child) while taking Ezmekly. Your doctor may decide to lower your dose, stop treatment temporarily or permanently:

• Eye problems

  Ezmekly can cause eye problems that can lead to blindness (see section 4 ‘Possible side effects’). A doctor (eye specialist) will have to check your vision (the vision of your child) before and regularly during treatment. Tell your doctor straight away if you get blurred vision, or any other changes to your vision during treatment.

• Heart problems

  Ezmekly can lower the amount of blood pumped by your heart (see section 4 ‘Possible side-effects’). In clinical studies of Ezmekly at the recommended dose, this did not cause any symptoms, however it can make you feel tired. Tell your doctor if you feel tired. Your doctor will check how well your heart works before and during your treatment with Ezmekly.

• Skin rash

  Skin rashes are very common with Ezmekly and can also become severe. Tell your doctor if you get a skin rash which may be bumpy or acne-like (see section 4 ‘Possible side effects’). Your doctor may provide guidance for managing your skin rash, including treatment (e.g., cream).

• Pregnancy

  Ezmekly is not recommended during pregnancy. Tell your doctor straight away if you or your partner becomes pregnant while taking this medicine. See ‘Pregnancy, breast-feeding and fertility’ section below.

Children younger than 2 years of age

Do not give Ezmekly to children younger than 2 years old. It has not been studied in this age group.

Other medicines and Ezmekly

Tell your doctor or pharmacist if you are taking, have recently taken or might take any other medicines, including herbal medicines while receiving Ezmekly.

Pregnancy, breast‑feeding and fertility

Ezmekly is not recommended during pregnancy. It may cause harm to your unborn baby.

If you are pregnant, think you may be pregnant or are planning to have a baby, ask your doctor for advice before taking this medicine. Your doctor may ask you to take a pregnancy test before starting treatment.

You or your partner should not become pregnant while taking this medicine. If you are able to become pregnant, you must use effective contraception (birth control). See ‘Contraception - information for women and men’ below.

If you or your partner become pregnant during treatment, tell your doctor straight away.

Contraception – information for women and men

If you are a woman who could become pregnant, you should use effective contraception while you are taking this medicine and for 6 months after the last dose. If you are a man whose partner could become pregnant, you should use effective contraception while you are taking this medicine and for 3 months after the last dose. It is not known if Ezmekly reduces how well hormonal contraceptives work. Please tell your doctor if you or your partner are taking a hormonal contraceptive, as your doctor may recommend using a second form of birth control (e.g. condoms).

Breast‑feeding

It is not known if Ezmekly passes into breast milk. A risk to the breast-fed child cannot be excluded. Do not breast-feed if you are taking Ezmekly and for one week after the last dose of Ezmekly.

Fertility (ability to have children)

Ezmekly may reduce fertility in men and women. You should discuss this with your doctor before starting treatment.

Driving and using machines

Ezmekly can cause side effects, including fatigue (feeling tired or weak) and blurred vision (see section 4 ‘Possible side-effects’), that may affect your ability to drive or use machines. Do not drive or use machines if you feel tired or if you have problems with your vision (such as blurred vision).

Ezmekly contains sodium

Each capsule contains less than 23 mg sodium (main component of cooking/table salt), that is to say it is essentially ‘sodium-free’.

3. How to take Ezmekly

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

How much to take

Your doctor will work out the correct dose for you (or your child) based on your height and weight. The maximum recommended dose is 8 mg a day (4 mg every 12 hours). The doctor will tell you how many capsules of Ezmekly to take.

How to take

• Take Ezmekly by mouth twice a day, about 12 hours apart. You should take this medicine every day for 21 days, and then stop taking it for 7 days. Each 28-day period is called a treatment cycle.

• Take Ezmekly with or without food.

• Swallow the capsules whole with water.
• Do not chew, break or open the capsules
• You can continue taking this medicine in treatment cycles until your disease gets worse or side effects become unacceptable. If you get side effects, your doctor may decide to lower your dose, stop treatment temporarily or permanently (see section 2 ‘Warnings and precautions’).
• Ezmekly is also available as a dispersible tablet. Your doctor will determine the correct formulation for you.

If you (or your child) are sick after taking Ezmekly

If you are sick (vomit) at any time after taking Ezmekly, do not take an extra dose. Take the next dose at the normal time.

If you (or your child) take more Ezmekly than you should

If you have taken more Ezmekly than you should, contact your doctor or pharmacist immediately.

If you (or your child) forget to take Ezmekly

If you forget to take a dose of Ezmekly, skip the missed dose and then take the next dose at the normal time. Do not take a double dose to make up for a forgotten dose.

If you have any further questions on the use of this medicine, ask your doctor or pharmacist.

If you (or your child) stop taking Ezmekly

Do not stop taking Ezmekly unless your doctor tells you as it may worsen your condition.

4. Possible side effects

Like all medicines, this medicine can cause side effects, although not everybody gets them. Contact your doctor if you experience any side effects. Your doctor may pause treatment with Ezmekly until your symptoms improve and/or they may reduce the dose you receive (see Section 3 “How to take”).

Serious side effects

Eye problems (common: may affect up to 1 in 10 people)

Ezmekly can cause eye problems in adults and children. Some of these eye problems may lead to a blockage of the vein draining to the eye (retinal vein occlusion or detachment of different layers of the eye (retinal pigment epithelial detachment)). Tell your doctor straight away if you get blurred vision or any other changes to your sight during treatment. Your doctor may ask you to stop taking this medicine or send you to a specialist, if you develop symptoms that include:

• blurred vision
• other changes to your vision (such as reduced vision, coloured dots in your vision)

Tell your doctor straight away if you notice any of the serious side effects above.

Other side effects

Talk to your doctor if you get any of the following side effects.

Adults

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

• skin rash with a flat discoloured area or raised acne-like bumps (dermatitis acneiform)
• diarrhoea
• increased blood levels of creatine phosphokinase, an enzyme found mainly in heart, brain and skeletal muscle
• feeling sick (nausea)
• being sick (vomiting)
• pain in muscles or bone
• feeling tired, weak or lacking energy (fatigue)
• stomach (abdominal) pain
• constipation
• rash
• headache
• increased levels of liver enzymes, as shown in blood tests
• swelling of the hands or feet (oedema peripheral)
• a decrease in the amount of blood that the heart is pumping (decreased left ventricular ejection fraction)
• dry skin
• itching
• hair loss or thinning (alopecia)

Package leaflet: Information for the patient

Ezmekly 1 mg hard capsules

Ezmekly 2 mg hard capsules

mirdametinib

Ezmekly® 1 mg hard capsules Ezmekly® 2 mg hard capsules

mirdametinib

Ezmekly® 1 mg hard capsules

Ezmekly® 2 mg hard capsules

mirdametinib

D2_v2_0000_EZMEKLY_UK_CAPSULES_PIL 18 August 2026

2XXXXXXXXX

2XXXXXXXXX

3.175mm between text and trim

349mm

rt_TEMPLATE - TOP BACK Width: 349mm, Length: 423mm

Fold Size: 72mm x 36mm

3.175mm between text and trim Common (may affect up to 1 in 10 people):

• decreased blood levels of neutrophils and leukocytes (types of white blood cells that help fight infections)
• mouth sores (stomatitis)
• dry mouth
• infection around the nails (paronychia)
• dry or itchy skin rash (eczema)
• changes in hair color
• changes in hair texture

Paediatrics

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

• increased blood levels of creatine phosphokinase, an enzyme found mainly in heart, brain, and skeletal muscle
• diarrhoea
• skin rash with a flat discoloured area or raised acne-like bumps (dermatitis acneiform)
• pain in muscles or bone
• stomach (abdominal) pain
• feeling sick (nausea)
• being sick (vomiting)
• a decrease in the amount of blood that the heart is pumping (decreased ejection fraction)
• headache
• rash
• infection around the nails (paronychia)
• decreased blood levels of neutrophils and leukocytes (types of white blood cells that help fight infections)
• mouth sores (stomatitis)
• increased levels of liver enzymes as shown in blood tests
• dry skin
• hair loss or thinning (alopecia)
• dry or itchy skin rash (eczema)
• itching
• changes in hair color
• feeling tired, weak or lacking energy (fatigue)
• constipation

Common (may affect up to 1 in 10 people):

• changes in hair texture
• swelling of the hands or feet (oedema peripheral)

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 Yellow Card Scheme Website: www.mhra.gov.uk/yellowcard or search MHRA Yellow Card in the Google Play or Apple App Store.

5. How to store Ezmekly

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 packaging. The expiry date refers to the last day of that month.

Store below 30°C. Store in the original package to protect from light.

Do not throw away your medicine via wasterwater or household waste. Ask your pharmacist how to throw away medicines you no longer use. These measures will help protect the environment.

6. Contents of the pack and other information

What Ezmekly contains

The active substance is mirdametinib.

Ezmekly 1 mg hard capsules

Each hard capsule contains 1 mg of mirdametinib.

Ezmekly 2 mg hard capsules

Each hard capsule contains 2 mg of mirdametinib.

The other ingredients are:

Capsule content

Microcrystalline cellulose (E460)

Croscarmellose sodium (E468) (see section 2 ‘Ezmekly contains sodium’) Magnesium stearate (E572)

Capsule shell

Gelatin (E441)

Titanium dioxide (E171)

Yellow iron oxide (E172)

Brilliant blue (E133)

Printing ink

Potassium hydroxide (E525)

Propylene glycol (E1520)

Purified water

Shellac (E904)

Titanium dioxide (E171)

What Ezmekly looks like and contents of the pack

Ezmekly 1 mg hard capsules (capsules)

Capsule comprised of a light green opaque body and cap with ‘MIR 1 mg’ printed in white ink on the cap.

Ezmekly is packaged in plastic bottles, secured with child-resistant closures and aluminium foil induction seals.

Ezmekly 1 mg hard capsules are provided in a carton box containing one bottle of 42 capsules.

Not all pack sizes may be marketed.

Ezmekly 2 mg hard capsules (capsules)

Capsule comprised of a white opaque body and a blue-green opaque cap with ‘MIR 2 mg’ printed in white ink on the cap.

Ezmekly is packaged in plastic bottles, secured with child-resistant closures and aluminium foil induction seals.

Ezmekly 2 mg hard capsules are provided in a carton box containing one bottle of 42 or 84 capsules.

Not all pack sizes may be marketed.

Marketing Authorisation Holder

Merck Serono Ltd

5 New Square

Bedfont Lakes Business Park

Feltham

Middlesex

TW14 8HA

UK

an affiliate of Merck KGaA, Darmstadt, Germany

Manufacturer

MIAS Pharma Limited

Suite 1 First Floor, Stafford House

Strand Road, Portmarnock, D13 WC83

Ireland

Patheon France

40 Boulevard De Champaret

38300 Bourgoin Jallieu

France

This leaflet was last revised in 08/2026

This medicine has been given ‘conditional approval’. This means that there is more evidence to come about this medicine. The Agency will review new information on this medicine at least every year and this leaflet will be updated as necessary.

TW5557200