Orfadin

Country: European Union

Language: English

Source: EMA (European Medicines Agency)

Active ingredient:

nitisinone

Available from:

Swedish Orphan Biovitrum International AB

ATC code:

A16AX04

INN (International Name):

nitisinone

Therapeutic group:

Other alimentary tract and metabolism products,

Therapeutic area:

Tyrosinemias

Therapeutic indications:

Hereditary tyrosinemia type 1 (HT 1)Orfadin is indicated for the treatment of adult and paediatric (in any age range) patients with confirmed diagnosis of hereditary tyrosinemia type 1 (HT 1) in combination with dietary restriction of tyrosine and phenylalanine.Alkaptonuria (AKU)Orfadin is indicated for the treatment of adult patients with alkaptonuria (AKU).

Product summary:

Revision: 21

Authorization status:

Authorised

Authorization date:

2005-02-21

Patient Information leaflet

34
B. PACKAGE LEAFLET
35
PACKAGE LEAFLET: INFORMATION FOR THE USER
ORFADIN 2 MG HARD CAPSULES
ORFADIN 5 MG HARD CAPSULES
ORFADIN 10 MG HARD CAPSULES
ORFADIN 20 MG HARD CAPSULES
nitisinone
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, pharmacist or
nurse.
-
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, pharmacist or nurse.
This includes any possible
side effects not listed in this leaflet. See section 4.
WHAT IS IN THIS LEAFLET
1.
What Orfadin
is and what it is used for
2.
What you need to know before you take Orfadin
3.
How to take Orfadin
4.
Possible side effects
5.
How to store Orfadin
6.
Contents of the pack and other information
1.
WHAT ORFADIN IS AND WHAT IT IS USED FOR
Orfadin contains the active substance nitisinone. Orfadin is used to
treat:
-
a rare disease called hereditary tyrosinemia type 1 in adults,
adolescents and children (in any
age range)
-
a rare disease called alkaptonuria (AKU) in adults.
In these diseases your body is unable to completely break down the
amino acid tyrosine (amino acids
are building blocks of our proteins), forming harmful substances.
These substances are accumulated in
your body. Orfadin
blocks the breakdown of tyrosine and the harmful substances are not
formed.
For the treatment of hereditary tyrosinemia type 1, you must follow a
special diet while you are taking
this medicine, because tyrosine will remain in your body. This special
diet is based on low tyrosine
and phenylalanine (another amino acid) content.
For the treatment of AKU, your doctor may advise you to follow a
special diet.
2.
WHAT YOU NEED TO KNOW BEFORE YOU TAKE ORFADIN
DO NOT TAKE ORFADIN
-
if you are allergic to nitisinone or any of the other ingredients of
this medicine (listed

Summary of Product characteristics

1
ANNEX I
SUMMARY OF PRODUCT CHARACTERISTICS
2
1.
NAME OF THE MEDICINAL PRODUCT
Orfadin 2 mg hard capsules
Orfadin 5 mg hard capsules
Orfadin 10 mg hard capsules
Orfadin 20 mg hard capsules
2.
QUALITATIVE AND QUANTITATIVE COMPOSITION
Each capsule contains 2 mg nitisinone.
Each capsule contains 5 mg nitisinone.
Each capsule contains 10 mg nitisinone.
Each capsule contains 20 mg nitisinone.
For the full list of excipients, see section 6.1.
3.
PHARMACEUTICAL FORM
Hard capsule.
White, opaque capsules (6x16 mm) imprinted “NTBC 2mg” in black on
the body of the capsule.
White, opaque capsules (6x16 mm) imprinted “NTBC 5mg” in black on
the body of the capsule.
White, opaque capsules (6x16 mm) imprinted “NTBC 10mg” in black on
the body of the capsule.
White, opaque capsules (6x16 mm) imprinted “NTBC 20mg” in black on
the body of the capsule.
The capsules contain a white to off white powder.
4.
CLINICAL PARTICULARS
4.1
THERAPEUTIC INDICATIONS
Hereditary tyrosinemia type 1 (HT-1)
Orfadin is indicated for the treatment of adult and paediatric (in any
age range) patients with
confirmed diagnosis of hereditary tyrosinemia type 1 (HT-1) in
combination with dietary restriction of
tyrosine and phenylalanine.
Alkaptonuria (AKU)
Orfadin is indicated for the treatment of adult patients with
alkaptonuria (AKU).
4.2
POSOLOGY AND METHOD OF ADMINISTRATION
Posology
HT-1:
Nitisinone treatment should be initiated and supervised by a physician
experienced in the treatment of
HT-1 patients.
Treatment of all genotypes of the disease should be initiated as early
as possible to increase overall
survival and avoid complications such as liver failure, liver cancer
and renal disease. Adjunct to the
nitisinone treatment, a diet deficient in phenylalanine and tyrosine
is required and should be followed
by monitoring of plasma amino acids (see sections 4.4 and 4.8).
_ _
_Starting dose HT-1 _
The recommended initial daily dose in the paediatric and adult
population is 1 mg/kg body weight
administered orally. The dose of nitisinone shou