Orphacol

Country: European Union

Language: English

Source: EMA (European Medicines Agency)

Active ingredient:

cholic acid

Available from:

Theravia

ATC code:

A05AA03

INN (International Name):

cholic acid

Therapeutic group:

Bile acids and derivatives

Therapeutic area:

Digestive System Diseases; Metabolism, Inborn Errors

Therapeutic indications:

Orphacol is indicated for the treatment of inborn errors in primary bile-acid synthesis due to 3β-hydroxy-Δ5-C27-steroid oxidoreductase deficiency or Δ4-3-oxosteroid-5β-reductase deficiency in infants, children and adolescents aged one month to 18 years and adults.

Product summary:

Revision: 13

Authorization status:

Authorised

Authorization date:

2013-09-12

Patient Information leaflet

20
B. PACKAGE LEAFLET
21
PACKAGE LEAFLET: INFORMATION FOR THE USER
_ _
ORPHACOL 50 MG HARD CAPSULES
ORPHACOL 250 MG HARD CAPSULES
Cholic acid
This medicine is subject to additional monitoring. This will allow
quick identification of new safety
information. You can help by reporting any side effects you may get.
See the end of section 4 for how to
report side effects.
READ ALL OF THIS LEAFLET CAREFULLY BEFORE YOU START TAKING 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 or pharmacist.
-
This medicine has been prescribed for you. Do not pass it on to
others. It may harm them, even if
their signs of illness are the same as yours.
-
If you get any side effects, talk to your doctor or pharmacist. This
includes any possible side effects
not listed in this leaflet. See section 4.
_ _
WHAT IS IN THIS LEAFLET
:
1.
What Orphacol is and what it is used for
2.
What you need to know before you take Orphacol
3.
How to take Orphacol
4.
Possible side effects
5.
How to store Orphacol
6.
Contents of the pack and other information
1.
WHAT ORPHACOL IS AND WHAT IT IS USED FOR
Orphacol contains cholic acid, a bile acid which is normally produced
by the liver. Certain medical
conditions are caused by defects in bile acid production and Orphacol
is used to treat infants from one
month to 2 years of age, children, adolescents and adults with these
medical conditions. The cholic acid
contained in Orphacol replaces the bile acids that are missing due to
the defect in bile acid production.
2.
WHAT YOU NEED TO KNOW BEFORE YOU TAKE ORPHACOL
_ _
DO NOT TAKE ORPHACOL
-
if you are allergic to cholic acid or any of the other ingredients of
this medicine (listed in section 6).
-
if you are taking phenobarbital or primidone, a medicine to treat
epilepsy.
WARNINGS AND PRECAUTIONS
During your treatment, your doctor will carry out various blood and
urine tests at different time to see how
your body is handling this medicine and

Summary of Product characteristics

1
_ _
_ _
ANNEX I
SUMMARY OF PRODUCT CHARACTERISTICS
2
This medicinal product is subject to additional monitoring. This will
allow quick identification of new
safety information. Healthcare professionals are asked to report any
suspected adverse reactions. See
section 4.8 for how to report adverse reactions.
1.
NAME OF THE MEDICINAL PRODUCT
Orphacol 50 mg hard capsules
Orphacol 250 mg hard capsules
2.
QUALITATIVE AND QUANTITATIVE COMPOSITION
Orphacol 50 mg capsules
Each hard capsule contains 50 mg of cholic acid.
Orphacol 250 mg capsules
Each hard capsule contains 250 mg of cholic acid.
Excipient(s) with known effect: Lactose monohydrate (145.79 mg per
capsule of 50 mg and 66.98 mg per
capsule of 250 mg).
_ _
For the full list of excipients, see section 6.1.
3.
PHARMACEUTICAL FORM
Hard capsule (capsule).
Orphacol 50 mg capsules
Oblong, opaque, blue and white capsule.
Orphacol 250 mg capsules
Oblong, opaque, green and white capsule.
4.
CLINICAL PARTICULARS
4.1
THERAPEUTIC INDICATIONS
Orphacol is indicated for the treatment of inborn errors in primary
bile acid synthesis due to
3β-Hydroxy-Δ
5
-C
27
-steroid oxidoreductase deficiency or Δ
4
-3-Oxosteroid-5β-reductase deficiency in
infants, children and adolescents aged 1 month to 18 years and adults.
4.2
POSOLOGY AND METHOD OF ADMINISTRATION
Treatment must be initiated and monitored by an experienced
gastroenterologist/hepatologist or a
paediatric gastroenterologist/hepatologist in the case of paediatric
patients.
In case of persistent lack of therapeutic response to cholic acid
monotherapy, other treatment options
should be considered (see section 4.4). Patients should be monitored
as follows: 3-monthly during the first
year, 6-monthly during the subsequent three years and annually
thereafter (see below).
3
Posology
The dose must be adjusted for each patient in a specialised unit
according to blood and/or urine
chromatographic bile acid profiles.
_3β-Hydroxy-Δ_
_5_
_-C_
_27_
_-steroid oxidoreductase deficiency _
The daily dose ranges from 5 to 15 mg/kg in inf