Miglustat Gen.Orph 100 mg hard capsules – patient leaflet, side effects, dosage

1. What Miglustat Gen.Or

ph is and what it is used for

Miglustat Gen.Orph contains the act

ive substance miglustat which belongs to

a group of medicines that affect metabolis

m. It is used to treat two conditions:

• Miglustat Gen.Orph is

  used to treat mild to moderate typ

  e 1 Gaucher disease in adults.

In type 1 Gaucher disease, a substan in liver and spleen enlargement, cha

ce called glucosylceramide is not remove nges in the blood and bone disease.

d from your body. It starts to build up in c

ertain cells of the body’s immune system.

This can result

The usual treatment for type 1 Gau enzyme replacement therapy.

cher disease is enzyme replacement thera

py. Miglustat Gen.Orph is only used whe

n a patient is considered unsuitable for tr

eatment with

• Miglustat Gen.Orph is a

  lso used to treat progressive neur

  ological symptoms in Niemann-Pic

  k type C disease in adults and in ch

  ildren.

If you have Niemann-Pick type C dis eye movements, balance, swallowing

ease, fats such as glycosphingolipids build , and memory, and in seizures.

up in the cells of your brain. This can resu

lt in disturbances in neurological function

s such as slow

Miglustat Gen.Orph works by inhibi

ting the enzyme called ‘glucosylceramide

synthase’ which is responsible for the first

step in the synthesis of most glycosphingol

ipids.

1. 2. What you need to know

before you take Miglustat Gen.Or

ph

Do not take Miglustat Gen.Orph

• if you are allergic to miglusta

  t or any of the other ingredients of this m

  edicine (listed in section 6)

Warnings and precautions

Talk to your doctor or pharmacist be - if you suffer from kidney dis - if you suffer from liver diseas

fore taking Miglustat Gen.Orph ease

e

Your doctor will perform the followi

ng tests before treatment and during treat nerves in your arms and legs

ment with Miglustat Gen.Orph:

• an examination to check the - measurement of vitamin B12

  levels

• monitoring growth if you are - monitoring of blood platelet

  a child or adolescent with Niemann-Pick counts

  type C disease

The reason for these tests is that som help the doctor decide whether thes

e patients have had tingling or numbness e effects are due to your disease or other e

in the hands and feet, or a decrease in body xisting conditions, or due to side effects of

weight, while taking Miglustat Gen.Orph. Miglustat Gen.Orph (see section 4 for furt

The tests will her details).

If you have diarrhoea, your doctor Orph together with food, or to temp inflammatory disease affecting the g these measures, or if you have any ot is another cause of your symptoms.

may ask you to change your diet to reduce orarily reduce your dose. In some cases th ut) have been reported in patients with Ni her abdominal complaint, consult your d

your lactose and carbohydrate intake suc e doctor may prescribe anti-diarrhoeal me emann-Pick type C disease treated with Mi octor. In such case, your doctor may decid

h as sucrose (cane sugar), or not to take M dicines such as loperamide. Cases of Croh glustat Gen.Orph. If your diarrhoea does n e to conduct further investigations to deter

iglustat Gen. n’s disease (an ot respond to mine if there

Male patients should use reliable bir

th control methods during their treatmen

t with Miglustat Gen.Orph, and for 3 mon

ths after finishing treatment.

Children and adolescents

Do not give this medicine to childre

n and adolescents (below 18 years old) wit

h type 1 Gaucher disease because it is not

known if it works in this disease.

Other medicines and Miglustat Ge

n.Orph

Tell your doctor or pharmacist if you

are taking, have recently taken, or might

take any other medicines.

Tell your docto Gen.Orph in y

r if you are taking medicines containing im our body.

iglucerase, which are sometimes used at t

he same time as Miglustat Gen.Orph. The

y may lower the amount of Miglustat

Pregnancy, bre

ast-feeding and fertility

You should not while taking M

take Miglustat Gen.Orph if you are pregn iglustat Gen.Orph. Do not breastfeed whi

ant or thinking of becoming pregnant. Yo le you are taking Miglustat Gen.Orph.

ur doctor can give you more information.

You must use effective birth control

Male patients s

hould use reliable birth control methods d

uring their treatment with Miglustat Gen

.Orph, and for 3 months after finishing tre

atment.

If you are preg

nant, breast feeding, think you may be pre

gnant or planning to have a baby, ask you

r doctor or pharmacist for advice before ta

king this medicine.

Driving and u

sing machines

Miglustat Gen.

Orph may make you feel dizzy. Do not dr

ive or use any tools or machines if you fee

l dizzy.

Miglustat Gen

.Orph contains sodium

This medicine

contains less than 1 mmol sodium (23 mg

) per hard capsule, that is to say essentiall

y ‘sodium-free’.

3. How t

o take Miglustat Gen.Orph

Always take thi

s medicine exactly as your doctor has told

you. Check with your doctor or pharmac

ist if you are not sure.

• For typ

  e 1 Gaucher disease: for adults, the usual s (300 mg).

  capsule

  dose is one capsule (100 mg) three times a

  day (morning, afternoon and evening). Th

  is means a daily maximum of three

• For Nie

  mann-Pick type C disease: For adults a ). This means a daily maximum of six cap

  nd adolescents (over 12 years old), the us sules (600 mg).

  ual dose is two capsules (200 mg) three ti

  mes a day (morning, afternoon and evening

For children le

ss than 12 years old, your doctor will adju

st the dose for Niemann-Pick type C dise

ase.

If you have a pr from diarrhoea

oblem with your kidneys you may receive when taking Miglustat Gen.Orph (see se

a lower starting dose. Your doctor may r ction 4). Your doctor will tell you how lon

educe your dose, e.g., to one capsule (100 g your treatment will last.

mg) once or twice a day, if you suffer

Miglustat Gen.

Orph can be taken with or without food.

You should swallow the whole capsule wit

h a glass of water.

If you take mo

re Miglustat Gen.Orph than you should

If you take mor

e capsules than you were told to, consult y

our doctor immediately. Miglustat has bee

n used in clinical studies at doses up to 300

0 mg: this caused decreases in white blood cells and

other side effects similar to those describ

ed in section 4.

If you forget to

take Miglustat Gen.Orph

Take the next c

apsule at the usual time. Do not take a do

uble dose to make up for a forgotten dose.

If you stop tak

ing Miglustat Gen.Orph

Don’t stop taki If you have any

ng Miglustat Gen.Orph without talking to further questions on the use of this medi

your doctor.

cine, ask your doctor or pharmacist.

4. Possib

le side effects

Like all medici

nes, this medicine can cause side effects, a

lthough not everybody gets them.

Most serious si Some patients

de effects:

have had tingling or numbness in the ha

nds and feet (seen commonly). They cou will perform some tests before and during

ld be signs of peripheral neuropathy, due t treatment with Miglustat Gen.Orph to as

o side effects of Miglustat Gen.Orph sess this (see section 2).

or they could b

e due to existing conditions. Your doctor

If you do get a

ny of these effects, please seek medical a

dvice from your doctor as soon as possib

le.

If you get a sl

ight tremor, usually trembling hands, s

eek medical advice from your doctor a e dose or stop Miglustat Gen.Orph treatm

s soon as possible. The tremor often disa ent to stop the tremor.

ppears without needing to stop the treatment. Som

etimes your doctor will need to reduce th

Very common

effects (may affect more than 1 in 10 peo mon side effects are diarrhoea, flatulence (

ple)

The most com

wind), abdominal (stomach) pain, weight

loss and decreased appetite.

If you do lose s

ome weight when you start treatment wi

th Miglustat Gen.Orph don’t worry. Peopl

e usually stop losing weight as treatment g

oes on.

Common effec

ts (may affect up to 1 in 10 people) ffects of treatment include headache, dizzi usea (feeling sick), constipation and vom mptoms and thrombocytopenia could be

Common side e (heartburn), na neurological sy

ness, paraesthesia (tingling or numbness), iting, swelling or discomfort in the abdo due to the underlying disease.

abnormal coordination, hypoaesthesia (re men (stomach) and thrombocytopenia (re

duced sensation to touch), dyspepsia duced levels of blood platelets). The

Other possible

side effects are muscular spasms or weak

ness, fatigue, chills and malaise, depressio

n, difficulty sleeping, forgetfulness and les

s libido.

Most patients g side effects cau

et one or more of these side effects, usuall se problems, consult your doctor. He or sh

y at the start of treatment or at intervals du e may reduce the dose of Miglustat Gen.

ring treatment. Most cases are mild and di Orph or recommend other medicines to he

sappear quite quickly. If any of these lp control side effects.

Reporting of side effects

If you get any side effects, talk to you Scheme

r doctor or pharmacist. This includes any

possible side effects not listed in this leaflet

. You can also report side effects directly vi

a Yellow Card

Website: www.mhra.gov.uk/yellowca on the safety of this medicine.

rd or search for MHRA Yellow Card in th

e Google Play or Apple App Store. By repo

rting side effects you can help provide mor

e information

5. How to store Miglustat

Gen.Orph

Keep this medicine out of the sight a

nd reach of children.

Do not take this medicine after the e

xpiry date which is stated on the carton a

fter ‘EXP’. The expiry date refers to the last

day of that month.

This medicine does not require any s

pecial storage conditions.

Do not throw away any medicines vi environment.

a wastewater or household waste. Ask you

r pharmacist how to throw away medicine

s you no longer use. These measures will he

lp protect the

6. Contents of the pack an

d other information

What Miglustat Gen.Orph contain

s

• The active substance is miglu

  stat 100 mg.

• The other ingredients are so

  dium starch glycolate (type A), povidone

  (K30), magnesium stearate, gelatin, titani

  um dioxide (E171). See section 2 “Miglus

  tat Gen.Orph contains sodium”.

What Miglustat Gen.Orph looks li

ke and contents of the pack

Miglustat Gen.Orph 100 mg hard ca Pack size of 84 hard capsules in non-

psules are white opaque cap and body, har perforated blisters and 84x1 hard capsule

d gelatin capsules size 4 of 14. mm length. s in perforated unit dose blisters.

Marketing Authorisation Holder

Gen.Orph

185 Bureaux de la Colline

92213 Saint Cloud Cedex

France

Manufacturer

Centre Lab

ZA Granderaie

23000 Guéret

France

This leaflet was last revised in Nove

mber 2025.