Ravicti

Country: European Union

Language: English

Source: EMA (European Medicines Agency)

Active ingredient:

glycerol phenylbutyrate

Available from:

Immedica Pharma AB

ATC code:

A16AX09

INN (International Name):

glycerol phenylbutyrate

Therapeutic group:

Other alimentary tract and metabolism products,

Therapeutic area:

Urea Cycle Disorders, Inborn

Therapeutic indications:

Ravicti is indicated for use as adjunctive therapy for chronic management of patients with urea cycle disorders (UCDs) including deficiencies of carbamoyl phosphate-synthase-I (CPS), ornithine carbamoyltransferase (OTC), argininosuccinate synthetase (ASS), argininosuccinate lyase (ASL), arginase I (ARG) and ornithine translocase deficiency hyperornithinaemia-hyperammonaemia homocitrullinuria syndrome (HHH) who cannot be managed by dietary protein restriction and/or amino acid supplementation alone.Ravicti must be used with dietary protein restriction and, in some cases, dietary supplements (e.g., essential amino acids, arginine, citrulline, protein-free calorie supplements).

Product summary:

Revision: 16

Authorization status:

Authorised

Authorization date:

2015-11-26

Patient Information leaflet

26
B. PACKAGE LEAFLET
27
PACKAGE LEAFLET: INFORMATION FOR THE PATIENT
RAVICTI 1.1 G/ML ORAL LIQUID
glycerol phenylbutyrate
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 only. 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 RAVICTI is and what it is used for
2.
What you need to know before you take RAVICTI
3.
How to take RAVICTI
4.
Possible side effects
5.
How to store RAVICTI
6.
Contents of the pack and other information
1.
WHAT RAVICTI IS AND WHAT IT IS USED FOR
RAVICTI contains the active substance ‘glycerol phenylbutyrate’
which is used to treat six known
‘urea cycle disorders’ (UCDs) in adults and children. The UCDs
include deficiencies of certain liver
enzymes such as carbamoyl phosphate synthetase I (CPS), ornithine
carbamoyltransferase (OTC),
argininosuccinate synthetase (ASS), argininosuccinate lyase (ASL),
arginase I (ARG) and ornithine
translocase deficiency hyperornithinaemia-hyperammonaemia
homocitrullinuria syndrome (HHH).
RAVICTI must be combined with a diet reduced in protein intake, and in
some cases a diet with
supplements such as essential amino acids (arginine, citrulline,
protein-free calorie supplements).
ABOUT UREA CYCLE DISORDERS
•
In urea cycle disorders, the body cannot remove the nitrogen from the
protein that we eat.
•
Normally, the body turns the extra nitrogen in the protein into a
waste compound called
‘ammonia’. The liver then removes ammonia from the body through a
cycle called the ‘urea
cycle’.
•
In urea cycle disorders, the body is not able to produce enough liver
enzymes to remove

Summary of Product characteristics

1
ANNEX I
SUMMARY OF PRODUCT CHARACTERISTICS
_ _
_ _
2
1.
NAME OF THE MEDICINAL PRODUCT
RAVICTI 1.1 g/ml oral liquid
2.
QUALITATIVE AND QUANTITATIVE COMPOSITION
Each ml of liquid contains 1.1 g of glycerol phenylbutyrate. This
corresponds to a density of 1.1 g/ml.
3.
PHARMACEUTICAL FORM
Oral liquid.
Clear, colourless to pale yellow liquid.
4.
CLINICAL PARTICULARS
4.1
THERAPEUTIC INDICATIONS
RAVICTI is indicated for use as adjunctive therapy for chronic
management of patients with urea
cycle disorders (UCDs) including deficiencies of carbamoyl phosphate
synthetase I (CPS), ornithine
carbamoyltransferase (OTC), argininosuccinate synthetase (ASS),
argininosuccinate lyase (ASL),
arginase I (ARG) and ornithine translocase deficiency
hyperornithinaemia-hyperammonaemia
homocitrullinuria syndrome (HHH) who cannot be managed by dietary
protein restriction and/or
amino acid supplementation alone.
RAVICTI must be used with dietary protein restriction and, in some
cases, dietary supplements (e.g.,
essential amino acids, arginine, citrulline, protein-free calorie
supplements).
4.2
POSOLOGY AND METHOD OF ADMINISTRATION
RAVICTI should be prescribed by a physician experienced in the
management of UCDs.
Posology
RAVICTI must be used with dietary protein restriction and sometimes
dietary supplements (e.g.,
essential amino acids, arginine, citrulline, protein-free calorie
supplements) depending on the daily
dietary protein intake needed to promote growth and development.
The daily dose should be individually adjusted according to the
patient’s protein tolerance and the
daily dietary protein intake needed.
RAVICTI therapy may be required life long unless orthotopic liver
transplantation is elected.
_Adults and children _
The recommended dose for patients naïve to phenylbutyric acid and for
patients switching from
sodium phenylbutyrate or from sodium phenylacetate/sodium benzoate
injection to RAVICTI are
different.
The recommended total daily dose of RAVICTI is based on body surface
area and ranges from
4.5 ml/m
2
/day to 11.2 ml/