Pombiliti® 105 mg powder f concentrate for solution fo – patient leaflet, side effects, dosage

What it is used for

Pombiliti is always used with ano medicine called miglustat 65 mg capsules. It is very important tha read the package leaflet of miglu 65 mg hard capsules.

ther

hard

t you also stat

If you have any questions about medicines, please ask your doct

your

or or

pharmacist.

How Pombiliti works

People with Pompe disease have low levels of the enzyme acid alp glucosidase (GAA). This enzyme control levels of glycogen (a type carbohydrate) in the body.

ha-

helps of

In Pompe disease, high levels of build up in the muscles of the bo keeps muscles, such as the mus help you walk, the muscles unde lungs that help you breathe, and muscle, from working properly.

glycogen dy. This cles that r the

the heart

Pombiliti enters the muscle cells

that are

affected by Pompe disease. Whe cells, the medicine works like GA help break down glycogen and c levels.

n in the A to

ontrol its

1. 2. What you need to know are given Pombiliti

before you

You must not be given Po

mbiliti

• If you have ever had life-t hypersensitivity reactions ▪ cipaglucosidase alfa ▪

  hreatening to:

  miglustat

  • any of the other ingredi medicine (listed in sect

    ents of this ion 6).

• If a previous infusion had and could not be restarte threatening hypersensitiv

  to be stopped d due to life ity reactions.

Warnings and precaution

s

Talk to your doctor, pharma

cist, or nurse

before using Pombiliti.

Speak to your doctor or n immediately if these apply think it might apply to you o ever had any such reaction enzyme replacement therap

urse

to you, if you r if you have s with another y (ERT):

• allergic reactions, includi (a severe allergic reaction

  ng anaphylaxis ) – see section

  4 under ‘Possible side eff symptoms of life-threaten

  ects’, below for ing reactions. ion while you e or in the

• infusion-associated react are receiving the medicin few hours afterwards - se under ‘Possible side effe symptoms of life-threaten

  e section 4

  cts’, below for ing reactions.

Inform your doctor if you ha heart or lung disease. Thes may worsen during or imm your infusion with Pombiliti. nurse immediately if you ar shortness of breath, cough, irregular heartbeat or any o from these conditions.

ve a history of e conditions ediately after Tell a doctor or e experiencing rapid or

ther effects

Also tell your doctor if you your legs or widespread sw body, severe skin rash or fr passing water. Your doctor if your Pombiliti infusion sh and the doctor will give you medical treatment.

have swelling in elling of your othy urine when will decide

ould stop,

appropriate

Your doctor will also d continue receiving Po

ecide if you can mbiliti.

Pre-treatment medic

ations

Your doctor may give before you have Pom medicines include:

you other medicines biliti. These

• antihistamines and

  corticosteroids to

  prevent or help redu reactions.

  ce infusion-related

• antipyretics to redu

  ce fever.

Children and adoles

cents

This medicine should patients under the age This is because the eff

not be given to of 18 years old. ects of Pombiliti

in conjunction with mi group are not known.

glustat in this age

Other medicines and

Pombiliti

Tell a doctor or nurse have recently used, or other medicines. This obtained without a pre herbal medicines.

if you are using, will be using any includes medicines scription, including

Pregnancy and brea

st-feeding

If you are pregnant or you may be pregnant, have a baby, do not ta but talk to your doctor immediately for advic medicine.

breast-feeding, think or are planning to ke this medicine or pharmacist

e before using this

There is no experienc Pombiliti in combinati during pregnancy.

e with the use of on with miglustat

• You should not rece take miglustat 65 m you are pregnant. B doctor immediately think that you may b

  ive Pombiliti and / or g hard capsules if e sure to tell your if you get pregnant, e pregnant, or if you

  are planning to bec may be risks to the

  ome pregnant. There unborn baby.

• Pombiliti in combina should not be given breast-feeding. A d be made whether to stop breast-feeding

  tion with miglustat to women who are ecision will need to stop treatment or to .

  VV-LAB-003314 - V2.0

  Contraception

  and fertility

  Female patients must use reliable during and for 4 medicines.

  of childbearing potential birth control methods weeks after stopping both

  Driving and usi

  ng machines

  You may feel diz

  zy, sleepy, or have low

  blood pressure ( Pombiliti or pre-t happens, do not machines.

  hypotensive) after having reatment medicines. If this drive or use any tools or

  Pombiliti contai

  ns sodium

  This medicinal p sodium (main co

  roduct contains 10.5 mg mponent of cooking/table

  salt) in each vial. 0.52% of the rec daily dietary inta

  This is equivalent to ommended maximum ke of sodium for an adult.

3. How Pombilit

i is given

Pombiliti is given nurse. It is given This is called an

to you by a doctor or through a drip into a vein. intravenous infusion.

Talk to your doct treated at home. upon evaluation home infusion of side effects duri your home infusi the infusion and treatment.

or if you would like to be Your doctor will decide if it is safe for you to have Pombiliti. If you get any ng an infusion of Pombiliti, on staff member may stop start appropriate medical

Pombiliti should with miglustat. Y 65 mg capsules Do NOT use mig (different produc instructions and of miglustat 65 m

be used in conjunction ou can only use miglustat with cipaglucosidase alfa. lustat 100 mg capsules t). Follow your doctor’s read the package leaflet g hard capsules for their

recommended d

ose.

How much Pom

biliti is given

The amount of m be given is base recommended d of body weight.

edicine that you will d on your weight. The ose is 20 mg for each kg

When and given

for how long Pombiliti is

• You will b every oth capsules Pombiliti. of miglus informati

  e treated with Pombiliti once er week. Miglustat 65 mg are taken on the same day as Refer to the package leaflet tat 65 mg hard capsules for on on how to take miglustat.

• The cipa start 1 ho hard cap ▪ In the e infusio from ta

  glucosidase alfa infusion should ur after taking miglustat 65 mg sules.

  vent of a delay, the start of n should not exceed 3 hours king miglustat.

• The infus approxim

  ion of cipaglucosidase alfa lasts ately 4 hours.

Figure 1. D

ose timeline

Typical infusion Take miglustat

Start cipaglucosidase alf

a* time

Complete cipaglucosidase alfa

Hours

0

1 2

4 hours

5 3 4

6 7

4 hours

Begin fasting

Stop fasting

* The cipag

lucosidase alfa infusion should start 1 h

our after taking miglustat capsules.

In the eve

nt of infusion delay, the start of infusion

should not exceed 3 hours from taking mi

glustat.

Switching replaceme

from another enzyme nt therapy (ERT)

If you are c another ER

urrently being treated with T:

• Your doc other ER

  tor will tell you when to stop the T before starting Pombiliti. doctor when you completed

• Tell your

  your last

  dose.

If you are should

given more Pombiliti than you

If you have or bloated, have been your docto

difficulty breathing, feel swollen or your heart is racing, you may given too much Pombiliti; tell r straight away. Excessive rate

of infu symp body, heart body.

sion of Pombiliti could result in toms related to too much fluid in the such as shortness of breath, rapid rate, or widespread swelling of the

If you

miss your dose of Pombiliti

If you conta as po comb miglu

have missed an infusion, please ct your doctor or nurse as soon ssible to reschedule Pombiliti in ination with miglustat 24 hours after stat was last taken.

If you stop receiving Pombiliti

and needles (paraesthesia)

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

p

• Rapid heartbeat

your

• Reddening of the skin

ent.

• Low blood pressure
• Shortness of breath

4. Possible side effects

• Cough
• Diarrhoea

Like all medicines, this medicine can c

ause

• Feeling sick (nausea)

side effects, although not everybody g them.

ets

• Stomach pain
• Passing gas
• Bloating

Pombiliti is used with miglustat, and si effects can occur with either of these medicines. Side effects were mainly seen while patients were being infused with Pombiliti (infusion-related effects)

de

• Vomiting
• Hives
• Itchy skin
• Rash
• Excessive sweating

or shortly after. You must tell your doct immediately if you get an infusion-associated reaction or an allergic react Some of these reactions may become serious and life-threatening. Your doct may give you medicines before your infusion to prevent these reactions.

or

• Painful muscle contractions
• Muscle pain

ion.

• Muscle weakness
• Joint pain

or

• Tiredness
• Fever
• Chills
• Feeling uncomfortable in chest

Infusion-associated reactions

• Swelling in the body area wher

  e needle

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

was inserted

n-

• Pain

lty

• Swelling in the hands, feet, ank

  les, legs

ess,

• Rise in blood pressure

Uncommon (may affect up to 1 i people)

n 100

Allergic reactions

Allergic reactions may include sympto such as rash anywhere on the body,

ms

• Allergic reaction

• Cannot hold or maintain balan

  ce

puffy eyes, prolonged difficulty breathi cough, swelling of the lip, tongue, or throat, itchy skin, and hives.

ng, • Burning sensation

• Migraine
• Feeling of near fainting
• Unusual paleness of the skin

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

• Asthma
• Wheezing
• Headache

• Discomfort in mouth and throa

  t

• Swollen throat

Common (may affect up to 1 in 10 pe

ople)

• Indigestion

• Serious life-threatening allergic react (anaphylactic reaction)

  ion

• Pain or painful contractions in

  gullet

• Mouth pain or discomfort
• Feeling dizzy
• Swollen tongue
• Tremor
• Skin discolouration
• Feeling sleepy
• Swelling of the skin
• Taste disturbance

• Pain in the area between the hi

  p and rib

• Sensation like numbness, tingling, p

  ins,

• Muscle tiredness

  • Muscle stiffness
  • Weakness

  • Pain in the cheek, gums,

    lips, chin needle was

  • Pain in body area where inserted
  • Feeling generally unwell
  • Pain in chest
  • Swelling face

  • Changes in body temper

    ature

  • Scratch or damage to the

    skin

Reporting of side effects

If you get any side effects, t doctor or nurse. This includ side effects not listed in this

alk to your

es any possible leaflet. You

can also report side effects details below). By reporting you can help provide more the safety of this medicine.

directly (see side effects information on

Yellow Card Scheme Website: www.mhra.gov.uk or search for MHRA Yellow Google Play or Apple App

/yellowcard Card in the Store

5. How to store Pombiliti

Your doctor, pharmacist, or responsible for storing this disposing of any opened vi The following information is healthcare professionals.

nurse is

medicine and als correctly. intended for

Keep this medicine out of t reach of children.

he sight and

Do not use this medicine af date which is stated on the carton after the letters “EXP date refers to the last date

ter the expiry bottle and ”. The expiry of that month.

Unopened vials: Store in th (2°C - 8°C). Keep the vial in carton in order to protect fr

e refrigerator the outer

om light.

After dilution, an immediate recommended. However, st intravenous bag with Pomb demonstrated for 6 hours a

use is

orage of the iliti has been t 20°C - 25°C

and 24 hours at 2°C -

8°C.

Do not throw away an wastewater or househ pharmacist how to thr that you no longer use will help protect the e

y medicines via old waste. Ask your ow away medicines . These measures nvironment.

6. Contents of the pa information

ck and other

What Pombiliti conta

ins

The active substance alfa. One vial contains cipaglucosidase alfa. the solution in the vial

is cipaglucosidase 105 mg of

After reconstitution, contains 15 mg

of cipaglucosidase alf recommended final co cipaglucosidase alfa d intravenous bag range 4 mg/mL.

a per mL. The

ncentration of

iluted into the

s from 0.5 mg/mL to

The other ingredients

are:

• Sodium citrate dihy

  drate (E331)

• Citric acid monohyd

  rate (E330)

• Mannitol (E421)

• Polysorbate 80 (E43

  3)

What Pombiliti looks of the pack

like and contents

Pombiliti is a white to powder. After reconsti as a clear to opalesce

slightly yellowish tution, it appears nt, colourless to

slightly yellow solution particles, practically fr the form of white to tr The reconstituted solu diluted into an intrave

, free of foreign

ee of particles in anslucent particles. tion must be further nous bag for infusion.

Pombiliti is a powder f solution for infusion in

or concentrate for a vial.

Packs of 1 vial, 10 via

ls, or 25 vials

Not all pack sizes may

be marketed.

VV-LAB-003314 - V2.0

Marketing Auth

orisation Holder

Amicus Therape One Globeside Fieldhouse Lane Marlow, Bucking SL7 1HZ

utics UK Limited

hamshire

Manufacturer

Manufacturing P B.V.

ackaging Farmaca (MPF)

Neptunus 12, He Netherlands

erenveen, 8448CN,

This leaflet was

last revised in 11/2024

Other sources o

f information

The following inf healthcare profe

ormation is intended for ssionals only:

Instructions for

use – reconstitution,

dilution, and ad

ministration

Pombiliti must b water for injectio sodium chloride for injections an by intravenous in and dilution sho accordance with

e reconstituted with n, then diluted with 9 mg/mL (0.9%) solution d then administered fusion. Reconstitution uld be performed in good practice rules,

particularly for th

e respect of asepsis.

Because this me formation may o solution and fina Therefore, a 0.2-binding in-line fil administration. It

dicine is a protein, particle ccur in the reconstituted l diluted infusion bags. micron low protein

ter should be used for was demonstrated

that the use of a removes visible result in an appa activity.

0.2 micron in-line filter particles and does not rent loss of protein or

Determine the n reconstituted ba patient’s dose re remove the requ

umber of vials to be sed on the individual gimen (mg/kg) and ired vials from the

refrigerator room temp 30 minutes single use

in order to allow them to reach erature (approximately

). Each vial of Pombiliti is for only.

Use asepti

c technique.

Reconstitu

tion

Reconstitut Pombiliti w using a syri not larger t water for in addition do directly ont

e each 105 mg per vial of ith 7.2 mL water for injections nge with a needle diameter han 18 gauge. Add the

jections by slow drop-wise wn the side of the vial and not

o the lyophilised powder. Tilt

and roll eac swirl, or sh volume app colourless of foreign p particles in particles. P of the reco matter and

h vial gently. Do not invert, ake the vial. The extraction ears as a clear to opalescent, to slightly yellow solution, free articles, and practically free of the form of white to translucent erform an immediate inspection nstituted vials for particulate discolouration. Do not use

if upon imm particles ot above are solution is reconstitut

ediate inspection foreign her than those described observed, or if the reconstituted discoloured. The pH of the ed solution is approximately 6.0.

After recon promptly di

stitution it is recommended to lute the vials (see below).

Dilution

When reco reconstitut 15 mg cipa reconstitut withdrawal from each

nstituted as above, the ed solution in the vial contains glucosidase alfa per mL. The ed volume allows accurate of 7.0 mL (equal to 105 mg) vial. This should then be

further dilut the reconst vial, includi for the part the patient’ syringe wit than 18 ga concentrati

ed as follows: slowly withdraw ituted solution from each ng less than the 7.0 mL ial vial, until the volume for s dose is obtained using a h a needle diameter not larger uge. The recommended final on of cipaglucosidase alfa in

the in to 4 m infusi of sod soluti with r the re sodiu

fusion bags ranges from 0.5 mg/mL g/mL. Remove airspace within the on bag. Also remove an equal volume ium chloride 9 mg/mL (0.9%) on for injections, that will be replaced econstituted Pombiliti. Slowly inject constituted Pombiliti directly into the m chloride 9 mg/mL (0.9%) solution for inj infusi Do no infusi

ections. Gently invert or massage the on bag to mix the diluted solution. t shake or excessively agitate the on bag.

The fi admin as po

nal infusion solution should be istered as close to preparation time ssible.

Any u shoul local r

nused medicine or waste material d be disposed of in accordance with equirements.

Admi

nistration

The P after t

ombiliti infusion should start 1 hour aking miglustat capsules. In the event shoul miglu

of infusion delay, the start of infusion d not exceed 3 hours from taking stat.

The re Pomb admin intrav

commended dose regimen of iliti is 20 mg/kg of body weight istered once every other week as an enous infusion.

Infusi incre infusi 1 mg/ 2 mg/ no sig reacti 7 mg/

ons should be administered mentally. It is recommended that the on begin at an initial rate of kg/hr and be gradually increased by kg/hr every 30 minutes if there are ns of IARs (infusion-associated ons) until a maximum rate of kg/hr is reached.