Emcitate

Package leaflet: Information for the patient

Emcitate 350 micrograms dispersible tablets

tiratricolo
Please read this leaflet carefully before taking this medicine because it contains
important information for you.

• Keep this leaflet. You may need to read it again.
• If you have any questions, ask your doctor or nurse.
• This medicine has been prescribed for you only. Do not give it to other people, even if they have the same symptoms as yours, because it could be harmful.
• If you experience any adverse reactions, including those not listed in this leaflet, contact your doctor or nurse. See section 4.

Contents of this leaflet

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

1. What Emcitate is and what it is used for

Emcitate contains the active substance tiratricol, which belongs to a group of medicines called thyroid hormones.
Emcitate is used for the treatment of thyrotoxicosis in patients with MCT8 deficiency (Allan-Herndon-Dudley syndrome).
Thyrotoxicosis in MCT8 deficiency occurs because a protein in the body, called MCT8, does not function as it should. As a result, thyroid hormones cannot enter and exit the body's cells properly, which can cause problems in the body and brain. The active substance in Emcitate, tiratricol, is very similar to a natural thyroid hormone present in the body called T3. Unlike natural T3, tiratricol does not require MCT8 to enter and exit cells, helping to restore the different types of thyroid hormones in the body to appropriate levels.

2. What you need to know before taking Emcitate

Do not take Emcitate

• if you are allergic to tiratricol or any of the other ingredients of this medicine (listed in section 6);
• if you have a condition called hyperthyroidism (an overactive thyroid gland) that is not caused by MCT8 deficiency;
• if you are pregnant (see section 2, Pregnancy and breastfeeding).

Do not take Emcitate if any of the above apply to you. If you are unsure, consult your doctor or nurse before taking Emcitate.

Warnings and precautions

Talk to your doctor or nurse before taking Emcitate if:

• you have diabetes;
• you have heart problems;
• you have liver or kidney problems.

If any of the above apply to you (or if you are unsure), consult your doctor or nurse before taking Emcitate.

Emcitate may cause certain unwanted effects known as symptoms of hypermetabolism (see section 4, Possible side effects). If you experience any of these symptoms and they persist, your doctor may adjust the dose of Emcitate. However, do not change the dose without first consulting your doctor.

Emcitate must not be taken for weight loss. It may cause serious or potentially life-threatening side effects, especially if taken together with orlistat for weight loss.

Other medicines and Emcitate

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

Do not take Emcitate and inform your doctor or nurse if you are taking any of the following medicines:

• Medicines for thyroid disorders that act like thyroid hormones, such as levothyroxine, propylthiouracil, and carbimazole. Taking these substances together with Emcitate may excessively increase or decrease thyroid hormone levels.
• Psychostimulants (substances that increase brain activity), such as methylphenidate, amphetamines, and caffeine. Taking these together with Emcitate may increase heart rate and blood pressure.

If any of the above apply to you (or if you are unsure), do not take Emcitate and inform your doctor or nurse.

Some medicines must be taken either much earlier or later than Emcitate, as they may reduce the amount of Emcitate absorbed by the body. Talk to your doctor or pharmacist before taking Emcitate if you are taking any of the following medicines:

• Antacids
• Charcoal tablets
• Calcium- or iron-containing medicines
• Sucralfate, a medicine for stomach problems
• Sevelamer carbonate, a medicine for kidney problems. These must be taken at least 2 hours before or after taking Emcitate.
• Colestiramine, a medicine to lower cholesterol. Take Emcitate at least 1 hour before or 4 hours after.

If you are unsure whether any of the above apply to you, consult your doctor or pharmacist before taking Emcitate.

Also inform your doctor or pharmacist if you are taking:

• Antidiabetic medicines – Emcitate may reduce blood glucose levels. This means your doctor may need to adjust your antidiabetic medicine dose.
• Anticoagulants – Emcitate may enhance the effect of anticoagulant medicines. As a result, you may bleed more easily. The dose of these medicines may need to be adjusted.
• Certain medicines for epilepsy (such as phenytoin, carbamazepine, and phenobarbital) – These may increase the rate at which your body metabolizes Emcitate. Your doctor will likely need to monitor your blood T3 levels regularly if you start, change, or stop treatment with these medicines. A dose adjustment of Emcitate may be necessary.
• Proton pump inhibitors (such as omeprazole, esomeprazole, pantoprazole, rabeprazole, and lansoprazole) used to reduce stomach acid production – These may reduce the amount of Emcitate absorbed by the body. A dose adjustment of Emcitate may be necessary.
• Antimalarials (such as chloroquine, proguanil) – These may cause hypothyroidism when used with Emcitate. Your doctor will likely need to monitor your blood T3 levels regularly during and after treatment with antimalarials and may need to adjust the dose of Emcitate.
• Antibiotics (used to treat bacterial infections) such as rifampicin and rifabutin – A dose adjustment of Emcitate may be necessary.
• Anti-inflammatory medicines, corticosteroids (such as hydrocortisone), and painkillers (such as salicylates, acetylsalicylic acid or naproxen, phenylbutazone, and aspirin) – These may reduce Emcitate levels in the blood.
• Herbal medicines [such as St. John’s wort (Hypericum perforatum)] – These may increase the rate at which your body metabolizes Emcitate. Your doctor will likely need to monitor your blood T3 levels regularly if you start, change, or stop treatment with these products.
• Immunosuppressants, medicines used after organ transplantation (such as cyclosporine, everolimus, sirolimus, and tacrolimus) – Emcitate may alter the rate at which these medicines are metabolized by the body.
• Medicines to lower blood cholesterol (such as atorvastatin, lovastatin, and simvastatin) – Emcitate may alter the rate at which these medicines are metabolized by the body.
• If you are taking estrogens or estrogen-containing products (such as hormone replacement therapy, but not contraceptives), you may require a higher dose of tiratricol.
• The weight-loss medicine orlistat may reduce the amount of tiratricol absorbed by the body, which could lower thyroid hormone levels. If you are taking orlistat together with tiratricol, your doctor will likely need to monitor your thyroid function.

If any of the above apply to you (or if you are unsure), consult your doctor or nurse before taking Emcitate.

Pregnancy and breastfeeding

You must not take Emcitate if you are pregnant, as it is not known whether this medicine could be harmful to the unborn baby.

While taking Emcitate, you must not become pregnant. If you are of childbearing age, you must use an effective contraceptive method (used to prevent pregnancy) during treatment with Emcitate.

If you become pregnant while taking Emcitate, you must stop treatment immediately and inform your doctor without delay.

Talk to your doctor before taking this medicine if you are breastfeeding. It is not known whether the medicine passes into breast milk. You and your doctor must decide whether to stop breastfeeding or stop taking Emcitate, weighing the benefits of breastfeeding for the child against the benefits of Emcitate for you.

Driving and using machines

Emcitate does not affect your ability to drive, ride a bicycle, or use tools or machinery.

If you think this medicine is impairing your ability to drive or use machinery, do not drive, ride a bicycle, or use tools or machinery until your symptoms have improved.

Emcitate contains lactose

Emcitate contains lactose, a type of sugar. If your doctor has diagnosed you with an intolerance to certain sugars, contact them before taking this medicine.

3. How to take Emcitate

Take this medicine exactly as instructed by your doctor or pharmacist. If you have any doubts, consult your doctor, pharmacist, or nurse.
Treatment will be initiated and monitored by physicians experienced in managing patients with rare genetic disorders such as MCT8 deficiency.

Dosage of Emcitate
Your doctor will determine the most appropriate dosage for you. The dose of Emcitate depends on thyroid hormone levels and body weight.

• The dose will be increased every two weeks until T3 levels are appropriate for your condition.
• If you are taking a stable dose of Emcitate, your blood T3 levels will be monitored regularly. If levels change and are no longer suitable, your doctor may decide to adjust the dose.
• Do not change the dose without first consulting your doctor.

When to take Emcitate
When starting to take 2 or more tablets per day, spread the doses throughout the day, for example in the morning, at midday, and in the evening.

How to take Emcitate
Emcitate dispersible tablets must always be dispersed in water before administration. The dispersion can be administered orally or via a feeding gastrostomy tube.

Oral administration

1. Prepare the dispersion in a small glass. This glass must not be used for any other purpose.
   • Mix the tablet or tablets (no more than 4 tablets at once) in 30 mL of drinking water. Use only drinking water—do not use any other liquid.
   • If you need to take half a tablet, break the tablet along the score line in the center.
   • Stir with a teaspoon for 1 minute. The dispersion should appear as a white, cloudy mixture. The teaspoon must not be used for any other purpose.
2. Use a syringe to draw up the dispersion from the glass. The syringe must be used only for Emcitate.
3. Slowly and gently push the plunger to gradually release the medicine into the cheek and then swallow.
4. Then, add an additional 10 mL of drinking water to the glass to rinse any remaining medicine.
   • Stir with the teaspoon for about 5 seconds to ensure any residual dispersion is mixed.
   • Draw up the dispersion from the glass using the same syringe.
5. Take Emcitate as described in step 3.

Administration via feeding gastrostomy tube

1. Prepare the dispersion in a small glass. This glass must not be used for any other purpose.
   • Mix the tablet or tablets (no more than 4 tablets at once) in 30 mL of drinking water. Use only drinking water—do not use any other liquid.
   • If you need to take half a tablet, break the tablet along the score line in the center.
   • Stir with a teaspoon for 1 minute. The dispersion should appear as a white, cloudy mixture. The teaspoon must not be used for any other purpose.
2. Use a syringe to draw up the dispersion from the glass. The syringe must be used only for Emcitate.
3. Carefully read the instructions for using the feeding tube before administering Emcitate and use the tube exactly as prescribed.
4. Then, add an additional 10 mL of drinking water to the glass to rinse any remaining medicine.
   • Stir with the teaspoon for about 5 seconds to ensure any residual dispersion is mixed.
   • Draw up the dispersion from the glass using the same syringe.
5. Administer Emcitate as prescribed for the feeding tube.
6. Flush and clean the tube as prescribed. A water flush volume of 3 mL is recommended.

If you take more Emcitate than you should
If you take more Emcitate than prescribed, contact a doctor immediately or go to hospital.
Take the medicine package with you.
The following effects may occur: rapid heartbeat, sweating, body overheating, nervousness, difficulty sleeping, or diarrhea (signs of hypermetabolic state).

• Your doctor may reduce the dose or discontinue treatment.

If you forget to take Emcitate

• If you miss a dose, you may take it if more than 4 hours remain before the next scheduled dose.
• If the next dose is due within 4 hours, skip the missed dose and take the next dose at the usual time.
• Do not take a double dose to make up for a forgotten dose.

If you stop taking Emcitate
Do not stop taking Emcitate without consulting your doctor.
If you have any questions 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.
The use of this medicine may lead to the following side effects:
Common (may affect up to 1 in 10 people):

• feeling irritable
• anxiety
• nightmares
• diarrhoea
• excessive sweating (hyperhidrosis)

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

• sleep disorders (insomnia)
• increased heart rate (tachycardia)
• feeling of excessive heat (hyperthermia)

Emcitate may cause so-called hypermetabolism symptoms at the beginning of treatment or when the dose is adjusted. These symptoms usually do not last longer than a few days, but you must inform your doctor immediately if you experience symptoms that could be signs of hypermetabolism, such as feeling irritable, anxiety, nightmares, excessive sweating, sleep disturbances, feeling of excessive heat, increased heart rate, temporary increase in blood pressure, or diarrhoea.
See section 2, Warnings and precautions.
Inform your doctor or nurse if you notice any of the side effects listed above. Also inform them if you think you have any side effects not listed in this list.

Reporting of side effects
If you get any side effects, including those not listed in this leaflet, talk to your doctor or nurse. You can also report side effects directly via the national reporting system referred to in Annex V. By reporting side effects, you can help provide more information on the safety of this medicine. Like all medicines, this medicine can cause side effects, although not everybody gets them.

5. How to store Emcitate

Keep this medicine out of the sight and reach of children.
Do not use this medicine after the expiry date stated on the carton and blister. The expiry date refers to the last day of that month.
Store in a refrigerator (2 °C-8 °C).
Keep in the original packaging to protect the medicine from light.

After dispersion:
the 30 mL dispersion can be stored at a temperature below 25 °C for up to 4 hours in the glass, and then re-suspended by stirring for 1 minute with a teaspoon before administration.

Do not dispose of any medicine via wastewater or household waste. Ask your pharmacist how to dispose of medicines you no longer use. This will help protect the environment.

6. Package contents and other information

What Emcitate contains

• Each tablet contains 350 micrograms of the active substance tiratricin.
• The other ingredients are monohydrate lactose (see section 2 “Emcitate contains lactose”), calcium hydrogen phosphate, maize starch, and magnesium stearate.

Description of the appearance of Emcitate and package contents
Emcitate is a white, oval tablet (dimensions: 10 mm in length, 5 mm in width) with score lines on both sides.
Emcitate is supplied in PVC/aluminum blisters, packed into an outer carton.
Emcitate is available in pack sizes containing 60 dispersible tablets.
Marketing Authorisation Holder
Rare Thyroid Therapeutics International AB
Klara Norra Kyrkogata 26
111 22 Stockholm
Sweden
Manufacturer
Cenexi
17 Rue De Pontoise
95529 Osny
France
Other 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. In addition, links to other websites on rare diseases and their therapeutic treatments are provided.