Sapropterin Dipharma

Country: European Union

Language: English

Source: EMA (European Medicines Agency)

Active ingredient:

Sapropterin dihydrochloride

Available from:

Dipharma Arzneimittel GmbH

ATC code:

A16AX07

INN (International Name):

sapropterin

Therapeutic group:

Other alimentary tract and metabolism products,

Therapeutic area:

Phenylketonurias

Therapeutic indications:

Sapropterin Dipharma is indicated for the treatment of hyperphenylalaninaemia (HPA) in adults and paediatric patients of all ages with phenylketonuria (PKU) who have been shown to be responsive to such treatment.Sapropterin Dipharma is also indicated for the treatment of hyperphenylalaninaemia (HPA) in adults and paediatric patients of all ages with tetrahydrobiopterin (BH4) deficiency who have been shown to be responsive to such treatment.

Product summary:

Revision: 3

Authorization status:

Authorised

Authorization date:

2022-02-16

Patient Information leaflet

40
B. PACKAGE LEAFLET
41
PACKAGE LEAFLET: INFORMATION FOR THE USER
SAPROPTERIN DIPHARMA 100 MG SOLUBLE TABLETS
sapropterin dihydrochloride
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 Sapropterin Dipharma is and what it is used for
2.
What you need to know before you take Sapropterin Dipharma
3.
How to take Sapropterin Dipharma
4.
Possible side effects
5.
How to store Sapropterin Dipharma
6.
Contents of the pack and other information
1.
WHAT SAPROPTERIN DIPHARMA IS AND WHAT IT IS USED FOR
Sapropterin Dipharma contains the active substance sapropterin which
is a synthetic copy of a body’s
own substance called tetrahydrobiopterin (BH4). BH4 is required by the
body to use an amino acid
called phenylalanine in order to build another amino acid called
tyrosine.
Sapropterin Dipharma is used to treat hyperphenylalaninaemia (HPA) or
phenylketonuria (PKU) in
patients of all ages. HPA and PKU are due to abnormally high levels of
phenylalanine in the blood
which can be harmful. Sapropterin Dipharma reduces these levels in
some patients who respond to
BH4 and can help increase the amount of phenylalanine that can be
included in the diet.
This medicine is also used to treat an inherited disease called BH4
deficiency in patients of all ages, in
which the body cannot produce enough BH4. Because of very low BH4
levels phenylalanine is not
used properly and its levels rise, resulting in harmful effects. By
replacing the BH4 that the body
cannot produce, Sapropterin Dipharma reduces the harmful excess of
phen

Summary of Product characteristics

1
ANNEX I
SUMMARY OF PRODUCT CHARACTERISTICS
2
1.
NAME OF THE MEDICINAL PRODUCT
Sapropterin Dipharma 100 mg soluble tablets
2.
QUALITATIVE AND QUANTITATIVE COMPOSITION
Each soluble tablet contains 100 mg of sapropterin dihydrochloride
equivalent to 77 mg of
sapropterin.
For the full list of excipients, see section 6.1.
3.
PHARMACEUTICAL FORM
Soluble tablet.
White to off-white, approximately 10 mm x 3.65 mm, round tablet
debossed with “11” on one side and
score line on the other side.
The score line is not intended for breaking the tablet.
4.
CLINICAL PARTICULARS
4.1
THERAPEUTIC INDICATIONS
Sapropterin Dipharma is indicated for the treatment of
hyperphenylalaninaemia (HPA) in adults and
paediatric patients of all ages with phenylketonuria (PKU) who have
been shown to be responsive to
such treatment (see section 4.2).
Sapropterin Dipharma is also indicated for the treatment of
hyperphenylalaninaemia (HPA) in adults
and paediatric patients of all ages with tetrahydrobiopterin (BH4)
deficiency who have been shown to
be responsive to such treatment (see section 4.2).
4.2
POSOLOGY AND METHOD OF ADMINISTRATION
Treatment with sapropterin dihydrochloride must be initiated and
supervised by a physician
experienced in the treatment of PKU and BH4 deficiency.
Active management of dietary phenylalanine and overall protein intake
while taking this medicinal
product is required to ensure adequate control of blood phenylalanine
levels and nutritional balance.
As HPA due to either PKU or BH4 deficiency is a chronic condition,
once responsiveness is
demonstrated, Sapropterin Dipharma is intended for long-term use (see
section 5.1).
Posology
_PKU _
The starting dose of sapropterin dihydrochloride in adult and
paediatric patients with PKU is 10 mg/kg
body weight once daily. The dose is adjusted, usually between 5 and 20
mg/kg/day, to achieve and
maintain adequate blood phenylalanine levels as defined by the
physician.
_BH4 deficiency _
The starting dose of sapropterin dihydrochloride in adult and
paediatric patients with BH4 def