Document Details

Generated Narrative: Bundle TEST PURPOSES ONLY - pombiliti

Document Subject

Generated Narrative: MedicinalProductDefinition mp639cce8c495cbcbb29789688f0c06663

identifier: http://ema.europa.eu/identifier/EU/1/22/1714/001

type: Medicinal Product

domain: Human use

status: active

legalStatusOfSupply: Medicinal product subject to medical prescription

name

productName: Pombiliti 105 mg powder for concentrate for solution for infusion

type: Full name

part

part: nan

type: Invented name part

part

part: nan

type: Scientific name part

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part: nan

type: Strength part

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part: nan

type: Pharmaceutical dose form part

Usages

-	Country	Jurisdiction	Language
*	EU	EU	en

Document Content

Generated Narrative: Composition composition-en-639cce8c495cbcbb29789688f0c06663

identifier: http://ema.europa.eu/identifier/EU/1/22/1714/001

status: Final

type: Package Leaflet

category: Raw

date: 2022-02-16 13:28:17+0000

author: Organization ACME industry

title: TEST PURPOSES ONLY - pombiliti

Attesters

What is in this leaflet

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

What Pombiliti is Pombiliti is a type of enzyme-replacement therapy (ERT) that is used in the treatment of late-onset Pompe disease in adults. It contains the active substance cipaglucosidase alfa .

What it is used for Pombiliti is always used with another medicine called miglustat 65 mg hard capsules. It is very important that you also read the package leaflet of miglustat 65 mg hard capsules.

If you have any questions about your medicines, please ask your doctor or pharmacist.

How Pombiliti works People with Pompe disease have low levels of the enzyme acid alpha-glucosidase (GAA). This enzyme helps control levels of glycogen (a type of carbohydrate) in the body.

In Pompe disease, high levels of glycogen build up in the muscles of the body. This keeps muscles, such as the muscles that help you walk, the muscles under the lungs that help you breathe, and the heart muscle, from working properly.

Pombiliti enters the muscle cells that are affected by Pompe disease. When in the cells, the medicine works like GAA to help break down glycogen and control its levels.

You must not be given Pombiliti

• If you have ever had life-threatening hypersensitivity reactions to:

cipaglucosidase alfa

miglustat

any of the other ingredients of this medicine (listed in section 6).

• If a previous infusion had to be stopped and could not be restarted due to life threatening hypersensitivity reactions.

Warnings and precautions Talk to your doctor, pharmacist, or nurse before using Pombiliti.

Speak to your doctor or nurse immediately if these apply to you, if you think it might apply to you or if you have ever had any such reactions with another enzyme replacement therapy (ERT):

• allergic reactions, including anaphylaxis (a severe allergic reaction) see section 4 under Possible side effects , below for symptoms of life-threatening reactions.
• infusion-associated reaction while you are receiving the medicine or in the few hours afterwards - see section 4 under Possible side effects , below for symptoms of life-threatening reactions.

Inform your doctor if you have a history of heart or lung disease. These conditions may worsen during or immediately after your infusion with Pombiliti. Tell a doctor or nurse immediately if you are experiencing shortness of breath, cough, rapid or irregular heartbeat or any other effects from these conditions.

Also tell your doctor if you have swelling in your legs or widespread swelling of your body, severe skin rash or frothy urine when passing water. Your doctor will decide if your Pombiliti infusion should stop, and the doctor will give you appropriate medical treatment. Your doctor will also decide if you can continue receiving Pombiliti.

Pre-treatment medications Your doctor may give you other medicines before you have Pombiliti. These medicines include:

• antihistamines and corticosteroids to prevent or help reduce infusion-related reactions.
• antipyretics to reduce fever.

Children and adolescents This medicine should not be given to patients under the age of 18 years old. This is because the effects of Pombiliti in conjunction with miglustat in this age group are not known.

Other medicines and Pombiliti Tell a doctor or nurse if you are using, have recently used, or will be using any other medicines. This includes medicines obtained without a prescription, including herbal medicines.

Pregnancy and breast-feeding If you are pregnant or breast-feeding, think you may be pregnant, or are planning to have a baby, do not take this medicine but talk to your doctor or pharmacist immediately for advice before using this medicine.

There is no experience with the use of Pombiliti in combination with miglustat during pregnancy.

• You should not receive Pombiliti and / or take miglustat 65 mg hard capsules if you are pregnant. Be sure to tell your doctor immediately if you get pregnant, think that you may be pregnant, or if you are planning to become pregnant. There may be risks to the unborn baby.
• Pombiliti in combination with miglustat should not be given to women who are breast-feeding. A decision will need to be made whether to stop treatment or to stop breast-feeding.

Contraception and fertility Female patients of childbearing potential must use reliable birth control methods during and for 4 weeks after stopping both medicines.

Driving and using machines You may feel dizzy, sleepy, or have low blood pressure (hypotensive) after having Pombiliti or pre-treatment medicines. If this happens, do not drive or use any tools or machines. Pombiliti contains sodium This medicinal product contains 10.5 mg sodium (main component of cooking/table salt) in each vial. This is equivalent to 0.52% of the recommended maximum daily dietary intake of sodium for an adult.

Pombiliti is given to you by a doctor or nurse. It is given through a drip into a vein. This is called an intravenous infusion. Talk to your doctor if you would like to be treated at home. Your doctor will decide upon evaluation if it is safe for you to have home infusion of Pombiliti. If you get any side effects during an infusion of Pombiliti, your home infusion staff member may stop the infusion and start appropriate medical treatment.

Pombiliti should be used in conjunction with miglustat. You can only use miglustat 65 mg capsules with cipaglucosidase alfa. Do NOT use miglustat 100 mg capsules (different product). Follow your doctor s instructions and read the package leaflet of miglustat 65 mg hard capsules for their recommended dose.

How much Pombiliti is given The amount of medicine that you will be given is based on your weight. The recommended dose is 20 mg for each kg of body weight. When and for how long Pombiliti is given

• You will be treated with Pombiliti once every other week. Miglustat 65 mg capsules are taken on the same day as Pombiliti. Refer to the package leaflet of miglustat 65 mg hard capsules for information on how to take miglustat.
• The cipaglucosidase alfa infusion should start 1 hour after taking miglustat 65 mg hard capsules.

In the event of a delay, the start of infusion should not exceed 3 hours from taking miglustat. The infusion of cipaglucosidase alfa lasts approximately 4 hours.

Figure 1. Dose timeline

• The cipaglucosidase alfa infusion should start 1 hour after taking miglustat capsules. In the event of infusion delay, the start of infusion should not exceed 3 hours from taking miglustat.

Switching from another enzyme replacement therapy (ERT) If you are currently being treated with another ERT:

• Your doctor will tell you when to stop the other ERT before starting Pombiliti.
• Tell your doctor when you completed your last dose.

If you are given more Pombiliti than you should If you have difficulty breathing, feel swollen or bloated, or your heart is racing, you may have been given too much Pombiliti; tell your doctor straight away. Excessive rate of infusion of Pombiliti could result in symptoms related to too much fluid in the body, such as shortness of breath, rapid heart rate, or widespread swelling of the body.

If you miss your dose of Pombiliti If you have missed an infusion, please contact your doctor or nurse as soon as possible to reschedule Pombiliti in combination with miglustat 24 hours after miglustat was last taken.

If you stop receiving Pombiliti Speak to your doctor if you wish to stop Pombiliti treatment. The symptoms of your disease may worsen if you stop treatment.

Like all medicines, this medicine can cause side effects, although not everybody gets them.

Pombiliti is used with miglustat, and side effects can occur with either of these medicines. Side effects were mainly seen while patients were being infused with Pombiliti (infusion-related effects) or shortly after. You must tell your doctor immediately if you get an infusion-associated reaction or an allergic reaction. Some of these reactions may become serious and life-threatening. Your doctor may give you medicines before your infusion to prevent these reactions.

Infusion-associated reactions Most infusion-associated reactions are mild or moderate. Symptoms of infusion-associated reaction may include difficulty breathing, bloating, fever, chills, dizziness, skin redness, itchy skin, and rash.

Allergic reactions Allergic reactions may include symptoms such as rash anywhere on the body, puffy eyes, prolonged difficulty breathing, cough, swelling of the lip, tongue, or throat, itchy skin, and hives.

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

• Headache

Common (may affect up to 1 in 10 people)

• Cough
• Sudden reddening of the face, neck, or upper chest
• Pain in chest
• Rash, itching
• Rise in blood pressure
• Sweating
• Bloating
• Passing gas or wind
• Loose, runny stools
• Vomiting
• Nausea
• Fever or chills
• Hives
• Swelling or pain in the body area where needle was inserted
• Muscle cramps, muscle pain, muscle weakness
• Involuntary shaking of one or more parts of the body
• Increased sweating
• Pain
• Altered sense of taste
• Feeling tired all the time, or feeling sleepy
• Shortness of breath

Uncommon (may affect up to 1 in 100 people)

• Breathing difficult and triggers coughing, a whistling sound (wheezing) when you breathe out, and shortness of breath (asthma)
• Allergic reaction
• Swelling in the hands, feet, ankles, legs
• Swelling of the skin
• Indigestion
• Belly pain
• Constant feeling of being tired
• Sore or irritated throat
• Painful and abnormal contractions of the throat
• Mouth irritation
• Mouth pain or discomfort in the back of the mouth
• Pain in the cheek, gums, lips, chin
• Loss of strength and energy, feeling weak
• Feeling of uneasiness, overall feeling of being sluggish
• Burning sensation
• Scratch or damage to the skin
• Changes in body temperature
• Decrease in a type of white blood cell shown in tests
• Feeling drowsy
• Feeling dizzy
• Pain in joints
• Pain in the area between the hip and rib
• Muscle fatigue
• Increased rigidity of muscles
• Cannot hold or maintain balance
• Low blood pressure
• Feeling of near fainting
• Pain in one or both sides of the head, throbbing pain, aura, eye pain, sensitivity to light (migraine)
• Skin discolouration

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 Appendix V. By reporting side effects you can help provide more information on the safety of this medicine.

Your doctor, pharmacist, or nurse is responsible for storing this medicine and disposing of any opened vials correctly. The following information is intended for healthcare professionals.

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 bottle and carton after the letters EXP . The expiry date refers to the last date of that month. Unopened vials: Store in the refrigerator (2 C - 8 C). Keep the vial in the outer carton in order to protect from light. After dilution, an immediate use is recommended. However, storage of the intravenous bag with Pombiliti has been demonstrated for 6 hours at 20 C - 25 C and 24 hours at 2 C - 8 C. Do not throw away any medicines via wastewater or household waste. Ask your pharmacist how to throw away medicines that you no longer use. These measures will help protect the environment.

What Pombiliti contains The active substance is cipaglucosidase alfa. One vial contains 105 mg of cipaglucosidase alfa. After reconstitution, the solution in the vial contains 15 mg of cipaglucosidase alfa per mL. The recommended final concentration of cipaglucosidase alfa diluted into the intravenous bag ranges from 0.5 mg/mL to 4 mg/mL.

The other ingredients are:

• Sodium citrate dihydrate (E331)
• Citric acid monohydrate (E330)
• Mannitol (E421)
• Polysorbate 80 (E433)

What Pombiliti looks like and contents of the pack

Pombiliti is a white to slightly yellowish powder. After reconstitution, it appears as a clear to opalescent, colourless to slightly yellow solution, free of foreign particles, practically free of particles in the form of white to translucent particles. The reconstituted solution must be further diluted into an intravenous bag for infusion.

Pombiliti is a powder for concentrate for solution for infusion in a vial

Packs of 1 vial, 10 vials, or 25 vials

Not all pack sizes may be marketed.

Marketing Authorisation Holder

Amicus Therapeutics Europe Limited Block 1, Blanchardstown Corporate Park Ballycoolin Road Blanchardstown, Dublin D15 AKK1 Ireland Tel: +353 (0) 1 588 0Fax: +353 (0) 1 588 6e-mail: info@amicusrx.co.uk

Manufacturer Manufacturing Packaging Farmaca (MPF) B.V. Neptunus 12, Heerenveen, 8448CN, Netherlands

For any information about this medicine, please contact the local representative of the Marketing Authorisation Holder:

Belgi /Belgique/Belgien Amicus Therapeutics Europe Limited T l/Tel: (+32) 0800 89e-mail: MedInfo@amicusrx.com

Lietuva Amicus Therapeutics Europe Limited
Tel: (+370) 8800 33El. pa tas: MedInfo@amicusrx.com

Amicus Therapeutics Europe Limited
Te .: (+359) 00800 111 3 : MedInfo@amicusrx.com

Luxembourg/Luxemburg Amicus Therapeutics Europe Limited
T l/Tel: (+352) 800 27e-mail: MedInfo@amicusrx.com esk republika Amicus Therapeutics Europe Limited
Tel.: (+420) 800 142 e-mail: MedInfo@amicusrx.com

Magyarorsz g Amicus Therapeutics Europe Limited
Tel.: (+36) 06 800 21e-mail: MedInfo@amicusrx.com Danmark Amicus Therapeutics Europe Limited
Tlf.: (+45) 80 253 e-mail: MedInfo@amicusrx.com

Malta Amicus Therapeutics Europe Limited
Tel: (+356) 800 62e-mail: MedInfo@amicusrx.com

Deutschland Amicus Therapeutics GmbH Tel: (+49) 0800 000 2E-Mail: MedInfo@amicusrx.com

Nederland Amicus Therapeutics BV Tel: (+31) 20 235 8510 / (+31) 0800 022 8e-mail: MedInfo@amicusrx.com Eesti Amicus Therapeutics Europe Limited
Tel: (+372) 800 0111 e-post: MedInfo@amicusrx.com

Norge Amicus Therapeutics Europe Limited
Tlf: (+47) 800 13e-post: MedInfo@amicusrx.com

Amicus Therapeutics Europe Limited
: (+30) 00800 126 e-mail: MedInfo@amicusrx.com

sterreich Amicus Therapeutics Europe Limited
Tel: (+43) 0800 909 E-Mail: MedInfo@amicusrx.com Espa a Amicus Therapeutics S.L.U. Tel: (+34) 900 941 e-mail: MedInfo@amicusrx.com

Polska Amicus Therapeutics Europe Limited
Tel.: (+48) 0080 012 15e-mail: MedInfo@amicusrx.com France Amicus Therapeutics SAS T l: (+33) 0 800 906 e-mail: MedInfo@amicusrx.com

Portugal Amicus Therapeutics Europe Limited
Tel: (+351) 800 812 e-mail: MedInfo@amicusrx.com Hrvatska Amicus Therapeutics Europe Limited
Tel: (+358) 0800 222 e-po ta: MedInfo@amicusrx.com

Ireland Rom nia Amicus Therapeutics Europe Limited
Tel.: (+40) 0808 034 e-mail: MedInfo@amicusrx.com

Slovenija Amicus Therapeutics Europe Limited
Tel: (+353) 1800 936 e-mail: MedInfo@amicusrx.com

Amicus Therapeutics Europe Limited
Tel.: (+386) 0800 81e-po ta: MedInfo@amicusrx.com

sland Amicus Therapeutics Europe Limited
S mi: (+354) 800 7Netfang: MedInfo@amicusrx.com Slovensk republika Amicus Therapeutics Europe Limited
Tel: (+421) 0800 002 e-mail: MedInfo@amicusrx.com

Italia Amicus Therapeutics S.r.l. Tel: (+39) 800 795 e-mail: MedInfo@amicusrx.com

Suomi/Finland Amicus Therapeutics Europe Limited
Puh/Tel: (+358) 0800 917 s hk posti/e-mail: MedInfo@amicusrx.com

Amicus Therapeutics Europe Limited
: (+357) 800 97e-mail: MedInfo@amicusrx.com

Sverige Amicus Therapeutics Europe Limited
Tfn: (+46) 020 795 e-post: MedInfo@amicusrx.com

Latvija Amicus Therapeutics Europe Limited
Tel: (+371) 800 05e-pasts: MedInfo@amicusrx.com

United Kingdom (Northern Ireland) Amicus Therapeutics, UK Limited
Tel: (+44) 08 0823 46e-mail: MedInfo@amicusrx.com

This leaflet was last revised in

Other sources of information Detailed information on this medicine is available on the European Medicines Agency web site: