Pombiliti – Package Leaflet: InfoCons – Consumer Protection presents, in an accessible format, the official package leaflet of the medicine Pombiliti, as authorised in the European Union and published in the Union Register of medicinal products.
Before anything else
Read all of this leaflet carefully before you are given this medicine because it contains important information for you.
- Keep this leaflet. You may need to read it again.
- If you have any further questions, ask your doctor, pharmacist, or nurse.
- If you get any side effects, talk to your doctor, pharmacist, or nurse. This includes any possible side effects not listed in this leaflet. See section 4.
ℹ️ 1. What Pombiliti is and what it is used for
What Pombiliti is Pombiliti is a type of ‘enzyme -replacement therap y’ (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 wit h 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 muscle s, 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.
⚠️ 2. What you need to know before you are given Pombiliti
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).
29 • If a previous infusion had to be stopped and could not be restarted due to life threatening hypersensi tivity 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 reaction s 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 imme diately 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 ur ine 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, hav e 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 risk s 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.
💊 3. How Pombiliti is given
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 the ir recommended dose.
How much Pombiliti is given The amount of medicine that you will be given is based on your weigh t. 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 cipagluc osidase alfa lasts approximately 4 hours.
Figure 1. Dos e 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.
31 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 l ast 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.
❗ 4. Possible side effects
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 be come serious an d life -threatening. Your doctor may give you medicines before your infusion to prevent these reactio ns.
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)
- Headac he
Common (may affect up to 1 in 10 people)
- Serious life -threatening allergic reaction (anaphylactic reaction)
- Feeling dizzy
- Tremor
- Feeling sleepy
- Taste disturbance
- Sensation like numbness, tingling, pins, and needles (paraesthesia)
- Rapid heartbeat
- Reddening of the skin
- Low blood pressure
- Shortness of breath
- Cough
- Diarrhoea
- Feeling sick (nausea)
- Stomach pain
- Passing gas
- Bloating
- Vomiting
- Hives 32 • Itchy skin
- Rash
- Excessive sweating
- Painful m uscle contractions
- Muscle pain
- Muscle weakness
- Joint pain
- Tiredness
- Fever
- Chills
- Feeling uncomfortable in chest
- Swelling in the body area where needle was inserted
- Pain
- Swelling in the hands, feet, ankles, legs
- Rise in blood pressure
Uncommon (may affect up to 1 in 100 people)
- Allergic reaction
- Cannot hold or maintain balance
- Burning sensation
- Migraine
- Feeling of near fainting
- Unusual paleness of the skin
- Asthma
- Wheezing
- Discomfort in mouth and throat
- Swollen throat
- Indigestion
- Pain or painful contractions in gullet
- Mouth pain or discomfort
- Swollen tongue
- Skin discolouration
- Swelling of the skin
- Pain in the area between the hip and rib
- Muscle tiredness
- Muscle stiffness
- Weakness
- Pain in the cheek, gums, lips, chin
- Pain in body area where needle was inserted
- Feeling generally unwell
- Pain in chest
- Swelling face
- Changes in body temperature
- Scratch or damage to the skin
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.
🌡️ 5. How to store Pombiliti
Your doctor, pharmacist , or nurse is responsible for storing this medicine and disposing of any opened vials correctly. The following information is intended for he althcare 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.
📦 6. Contents of the pack and other information
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.
34 Marketing Authorisation Holder Amicus Therapeutics Europe Limited Block 1, Blanchardstown Corporate Park Ballycoolin Road Blanchardstown, Dublin D15 AKK1 Ireland Tel: +353 (0) 1 588 0836 Fax: +353 (0) 1 588 6851 e-mail: [email protected]
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:
België/Belgique/Belgien Amicus Therapeutics Europe Limited Tél/Tel: (+32) 0800 89172 e-mail: [email protected] Lietuva Amicus Therapeutics Europe Limited Tel: (+370) 8800 33167 El. paštas : [email protected] България Amicus Therapeutics Europe Limited Teл.: (+359) 00800 111 3214 имейл: [email protected] Luxembourg/Luxemburg Amicus Therapeutics Europe Limited Tél/Tel: (+352) 800 27003 e-mail: [email protected] Česká republika Amicus Therapeutics Europe Limited Tel.: (+420) 800 142 207 e-mail: [email protected] Magyarország Amicus Therapeutics Europe Limited Tel.: (+36) 06 800 21202 e-mail: [email protected] Danmark Amicus Therapeutics Europe Limited Tlf.: (+45) 80 253 262 e-mail: [email protected] Malta Amicus Therapeutics Europe Limited Tel: (+356) 800 62674 e-mail: [email protected] Deutschland Amicus Therapeutics GmbH Tel: (+49) 0800 000 2038 E-Mail : [email protected] Nederland Amicus Therapeutics BV Tel: (+31) 20 235 8510 / (+31) 0800 022 8399 e-mail: [email protected] Eesti Amicus Therapeutics Europe Limited Tel: (+372) 800 0111 911 e-post: [email protected] Norge Amicus Therapeutics Europe Limited Tlf: (+47) 800 13837 e-post: [email protected] 35 Ελλάδα Amicus Therapeutics Europe Limited Τηλ: (+30) 00800 126 169 e-mail: [email protected] Österreich Amicus Therapeutics Europe Limited Tel: (+43) 0800 909 639 E-Mail : [email protected] España Amicus Therapeutics S.L.U.
Tel: (+34) 900 941 616 e-mail: [email protected] Polska Amicus Therapeutics Europe Limited Tel.: (+48) 0080 012 15475 e-mail: [email protected] France Amicus Therapeutics SAS Tél: (+33) 0 800 906 788 e-mail: [email protected] Portugal Amicus Therapeutics Europe Limited Tel: (+351) 800 812 531 e-mail: [email protected] Hrvatska Amicus Therapeutics Europe Limited Tel: (+ 385) 0800 222 452 e-pošta: [email protected] Ireland Amicus Therapeutics Europe Limited Tel: (+353) 1800 936 230 e-mail: [email protected] România Amicus Therapeutics Europe Limited Tel.: (+40) 800 476 561 e-mail: [email protected] Slovenija Amicus Therapeutics Europe Limited Tel.: (+386) 0800 81794 e-pošta: [email protected] Ísland Amicus Therapeutics Europe Limited Sími: (+354) 800 7634 Netfang : [email protected] Slovenská republika Amicus Therapeutics Europe Limited Tel: (+421) 0800 002 437 e-mail: [email protected] Italia Amicus Therapeutics S.r.l.
Tel: (+39) 800 795 572 e-mail: [email protected] Suomi/Finland Amicus Therapeutics Europe Limited Puh/Tel: (+358) 0800 917 780 sähköposti/e -mail: [email protected] Κύπρος Amicus Therapeutics Europe Limited Τηλ: (+357) 800 97595 e-mail: [email protected] Sverige Amicus Therapeutics Europe Limited Tfn: (+46) 020 795 493 e-post: [email protected] Latvija Amicus Therapeutics Europe Limited Tel: (+371) 800 05391 e-pasts : [email protected]
This leaflet was last revised in
Other sources of information Detailed information on this medicine is available on the European Medicines Agency web site: http://www.ema.europa.eu . There are also links to other websites about rare diseases and treatments.
🩺 Information intended for healthcare professionals only
Instructions for use – reconstitution, dilution, and administration Pombiliti must be reconstituted with water for injection, then diluted with sodium chloride 9 mg/mL (0.9%) solution for injections and then administered by intravenous infusion. Reconstitution and dilution should be performed in accordance with good practice rules, part icularly for the respect of asepsis.
Because this medicine is a protein, particle formation may occur in the reconstituted solution and final diluted infusion bags. Therefore, a 0.2 -micron low protein binding in -line filter should be used for administrati on. It was demonstrated that the use of a 0.2 micron in -line filter removes visible particles and does not result in an apparent loss of protein or activity.
Determine the number of vials to be reconstituted based on the individual patient’s dose regimen (mg/kg) and remove the required vials from the refrigerator in order to allow them to reach room temperature (approximately 30 minutes). Each vial of Pombiliti is for single use only.
Use aseptic technique.
Reconstitution Reconstitute each 105 mg per v ial of Pombiliti with 7.2 mL water for injections using a syringe with a needle diameter not larger than 18 gauge. Add the water for injections by slow drop -wise addition down the side of the vial and not directly onto the lyophilised powder. Tilt and roll each vial gently.
Do not invert, swirl, or shake the vial. The extraction volume appears as a clear to opalescent, colourless to slightly yellow solution, free of foreign particles, and practically free of particles in the form of white to translucent par ticles. Perform an immediate inspection of the reconstituted vials for particulate matter and discolouration. Do not use if upon immediate inspection foreign particles other than those described above are observed, or if the reconstituted solution is disco loured. The pH of the reconstituted solution is approximately 6.0.
After reconstitution it is recommended to promptly dilute the vials (see below).
Dilution When reconstituted as above, the reconstituted solution in the vial contains 15 mg cipaglucosida se alfa per mL. The reconstituted volume allows accurate withdrawal of 7.0 mL (equal to 105 mg) from each vial. This should then be further diluted as follows: Slowly withdraw the reconstituted solution from each vial, including less than the 7.0 mL for the partial vial, until the volume for the patient’s dose is obtained using a syringe with a needle diameter not larger than 18 gauge. The recommended final concentration of cipaglucosidase alfa in the infusion bags ranges from 0.5 mg/mL to 4 mg/mL.
Remove airspace within the infusion bag. Also remove an equal volume of sodium chloride 9 mg/mL (0.9%) solution for injections, that will be replaced with reconstituted Pombiliti. Slowly inject the reconstituted Pombiliti directly into the sodium chloride 9 mg/mL (0.9%) solution for injections.
Gently invert or massage the infusion bag to mix the diluted solution. Do not shake or excessively agitate the infusion bag.
The final infusion solution should be administered as close to preparation time as possible .
Any unused medicine or waste material should be disposed of in accordance with local requirements.
Administration The Pombiliti 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.
37 The recommended dose regimen of Pombiliti is 20 mg/kg of body weight administered once every other week as an intravenous infusion.
Infusions should be administered incrementally. It is recommended that the infusion begin at an initial rate of 1 mg/kg/hr and be gradually increased by 2 mg/kg/hr every 30 minutes if there are no signs of IARs (infusion -associated reactions) until a maximum rate of 7 mg/kg/hr is reached.
📄 Package Leaflet – official source
This article reproduces the information in the official package leaflet, as authorised in the European Union. You can consult the original document here: Product information Pombiliti (PDF, ec.europa.eu).
You can check this medicine at any time in the Union Register of medicinal products (EU number EU/1/22/1714).
⚠️ Important: This article is for information purposes only and does not replace medical advice or the package leaflet inside the medicine box. Read the leaflet carefully before use. For any questions about your treatment, talk to your doctor or pharmacist. If you experience side effects, tell your doctor or pharmacist and report them through your national reporting system.