MYOZYME

Nazione: Israele

Lingua: inglese

Fonte: Ministry of Health

Principio attivo:

ALGLUCOSIDASE ALFA

Commercializzato da:

SANOFI ISRAEL LTD

Codice ATC:

A16AB07

Forma farmaceutica:

POWDER FOR CONCENTRATE FOR INFUSION

Composizione:

ALGLUCOSIDASE ALFA 50 MG/VIAL

Via di somministrazione:

I.V

Tipo di ricetta:

Required

Prodotto da:

GENZYME EUROPE B.V., THE NETHERLANDS

Gruppo terapeutico:

ALGLUCOSIDASE ALFA

Area terapeutica:

ALGLUCOSIDASE ALFA

Indicazioni terapeutiche:

Myozyme is indicated for long-term enzyme replacement therapy (ERT) in patients with a confirmed diagnosis of pompe disease (acid alpha-glucosidase deficiency). The benefits of Myozyme in patients with late-onset Pompe disease have not been established.

Data dell'autorizzazione:

2021-11-30

Foglio illustrativo

העדוה
העדוה
לע
לע
הרמחה
הרמחה
(
(
עדימ
עדימ )תוחיטב )תוחיטב
ןולעב
ןולעב
אפורל
אפורל
:ךיראת
5.3.2014
םש
רישכת
:תילגנאב
MYOZYME
רפסמ :םושיר
135-74-31488-00
םש
לעב
:םושירה
SANOFI AVENTIS ISRAEL LTD
.
ספוט
הז
דעוימ
טוריפל
תורמחה
!דבלב
תורמחהה
תושקובמה
קרפ
ןולעב
טסקט
יחכונ
טסקט
שדח
4.4
SPECIAL
WARNINGS
AND
PRECAUTIONS
FOR USE
Immunogenicity
Transient nephrotic syndrome which resolved
following temporary interruption of ERT was
observed in one patient with infantile-onset
Pompe disease who received very frequent
dosing of rhGAA (10 mg/kg 5 times weekly)
over an extended period.
Immune-mediated reactions
Severe cutaneous reactions, possibly immune-
mediated, have been reported with alglucosidase
alfa, including ulcerative and necrotizing skin
lesions (see section 4.8).
Patients should be monitored for signs and
symptoms of systemic immune mediated reactions
involving skin and other organs while receiving
alglucosidase alfa. If immune-mediated reactions
occur, discontinuation of the administration of
alglucosidase alfa should be considered and
appropriate medical treatment initiated. The risks
and benefits of re-administering alglucosidase alfa
following an immune-mediated reaction should be
considered. Some patients have been successfully
rechallenged and continued to receive
alglucosidase alfa under close clinical supervision.
________
Immune-mediated reactions
Severe cutaneous reactions, possibly immune-
mediated, have been reported with alglucosidase
alfa, including ulcerative and necrotizing skin
lesions (see section 4.8).
Nephrotic syndrome was
observed in a few Pompe patients treated with
alglucosidase alfa and who had high IgG antibody
titres (≥ 102,400) (see section 4.8). In these
patients renal biopsy showed immune complex
deposition. Patients improved following treatment
interruption. It is therefore recommended to
perform periodic urinalysis

Scheda tecnica

1
MYO-IV-SPC-16.0
_ _
Myozyme
Powder for concentrate for solution for infusion.
1.
NAME OF THE MEDICINAL PRODUCT
_ _
Myozyme
50 mg powder for concentrate for solution for infusion.
2.
QUALITATIVE AND QUANTITATIVE COMPOSITION
One vial contains 50 mg of alglucosidase alfa.
After reconstitution, the solution contains 5 mg of alglucosidase
alfa* per ml and after dilution, the
concentration varies from 0.5 mg to 4 mg/ml.
*Human acid α-glucosidase is produced in Chinese hamster ovary cells
(CHO) by recombinant DNA
technology.
For the full list of excipients, see section 6.1.
3.
PHARMACEUTICAL FORM
Powder for concentrate for solution for infusion.
White to off-white powder.
4.
CLINICAL PARTICULARS
4.1
Therapeutic indications
Myozyme is indicated for long-term enzyme replacement therapy (ERT) in
patients with a confirmed
diagnosis of Pompe disease (acid

-glucosidase deficiency).
The benefits of Myozyme in patients with late-onset Pompe disease have
not been established.
4.2
Posology and method of administration
Myozyme treatment should be supervised by a physician experienced in
the management of patients
with Pompe disease or other inherited metabolic or neuromuscular
diseases.
Posology
The recommended dose regimen of alglucosidase alfa is 20 mg/kg of body
weight administered once
every 2 weeks.
Patient response to treatment should be routinely evaluated based on a
comprehensive evaluation of
all clinical manifestations of the disease.
_Paediatric and older people _
There is no evidence for special considerations when Myozyme is
administered to paediatric patients
of all ages or older people.
2
_Patients with renal and hepatic impairment_
The safety and efficacy of Myozyme in patients with renal or hepatic
impairment have not been
evaluated and no specific dose regimen can be recommended for these
patients.
Method of administration
Myozyme should be administered as an intravenous infusion.
Infusions should be administered incrementally. It is recommended that
the infusion begin at an initial
rate of 1 mg/kg/h and b