VIMIZIM

Country: New Zealand

Language: English

Source: Medsafe (Medicines Safety Authority)

Active ingredient:

Elosulfase alfa 1 mg/mL;  

Available from:

Pharmacy Retailing (NZ) Ltd t/a Healthcare Logistics

INN (International Name):

Elosulfase alfa 1 mg/mL

Dosage:

1 mg/mL

Pharmaceutical form:

Concentrate for injection

Composition:

Active: Elosulfase alfa 1 mg/mL   Excipient: Arginine hydrochloride Monobasic sodium phosphate monohydrate Polysorbate 20 Sodium acetate trihydrate Sorbitol Water for injection

Prescription type:

Prescription

Manufactured by:

BioMarin Pharmaceutical Inc

Therapeutic indications:

Treatment of mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome) in children and adults of all ages.

Product summary:

Package - Contents - Shelf Life: Vial, glass, - 5 mL - 36 months from date of manufacture stored at 2° to 8°C (Refrigerate, do not freeze) protect from light

Authorization date:

2015-02-19

Patient Information leaflet

VIMIZIM
®
1
NEW ZEALAND CONSUMER MEDICINE INFORMATION
VIMIZIM
®
_elosulfase alfa (rch) _
WHAT IS IN THIS LEAFLET
This leaflet answers some common
questions about Vimizim.
It does not contain all the available
information. It does not take the
place of talking to your doctor or
pharmacist.
All medicines have benefits and
risks. Your doctor has weighed the
risks of treating you with Vimizim
against the benefits expected for you.
IF YOU HAVE ANY CONCERNS ABOUT
RECEIVING THIS MEDICINE, TALK TO
YOUR DOCTOR, NURSE OR THE HOSPITAL
PHARMACIST.
KEEP THIS LEAFLET WHILE YOU ARE
BEING TREATED WITH VIMIZIM.
You may need to read it again.
WHAT VIMIZIM IS USED
FOR
Vimizim is used to treat children and
adults of all ages with MPS IVA
(Mucopolysaccharidosis Type IVA,
Morquio A Syndrome). People with
MPS IVA have either a low level, or
reduced activity, of an enzyme called
N-acetylgalactosamine-6-sulfatase
(or GALNS), which breaks down
specific substances (for example,
keratan sulfate) in the body. As a
result, these specific substances do
not get broken down and processed
by the body as they should. They
accumulate in many tissues in the
body, which causes the symptoms of
MPS IVA.
_HOW VIMIZIM WORKS _
This medicine contains an enzyme
called elosulfase alfa. Elosulfase alfa
is a recombinant version of a human
enzyme produced by genetic
engineering in Chinese Hamster
Ovary (CHO) cells.
It works by replacing the natural
enzyme in MPS IVA patients.
Treatment with VIMIZIM has shown
improvement in walking ability and
reduction of the levels of keratan
sulfate
.
ASK YOUR DOCTOR IF YOU HAVE ANY
QUESTIONS ABOUT WHY VIMIZIM HAS
BEEN PRESCRIBED FOR YOU.
Vimizim is available only with a
doctor's prescription.
_USE IN CHILDREN _
Vimizim is recommended for use in
children and adults.
BEFORE STARTING
TREATMENT WITH
VIMIZIM
_WHEN YOU MUST NOT HAVE IT _
DO NOT HAVE VIMIZIM IF YOU
EXPERIENCE SEVERE OR LIFE-
THREATENING ALLERGIC REACTIONS TO
ELOSULFASE ALFA OR ANY OF THE OTHER
INGREDIENTS OF VIMIZIM AND YOUR
DOCTOR IS NOT ABLE TO CONTROL THESE
RE

Summary of Product characteristics

Data Sheet - New Zealand
V4
Supersedes: V3 (14 August 2017)
Page 1 of 14
NEW ZEALAND DATA SHEET
1. VIMIZIM 1 MG/ML CONCENTRATED SOLUTION FOR INJECTION
VIMIZIM
®
elosulfase alfa (rch) 1 mg/mL concentrated solution for injection.
2. QUALITATIVE AND QUANTITATIVE COMPOSITION
Each mL of solution contains 1 mg elosulfase alfa. *Each vial of
approximately 5 mL extractable
solution contains 5 mg elosulfase alfa.
*Elosulfase alfa is a purified human enzyme produced by recombinant
DNA technology in a Chinese
hamster ovary cell line.
Human N-acetylgalactosamine-6-sulfatase (EC 3.1.6.4) is a lysosomal
enzyme that hydrolyses sulfate
from either galactose-6-sulfate or N-acetyl-galactosamine-6-sulfate on
the non-reducing ends of the
glycosaminoglycans keratan sulfate (KS) and chondroitin sulfate.
Elosulfase alfa is a soluble dimeric protein, and each monomer
contains 496 amino acids with an
approximate molecular mass of 55 kDa for the peptide chain. The
oligosaccharides present at the two
consensus N-linked glycosylation sites contain mannose-6-phosphate
(M6P). M6P is recognised by a
receptor at the cell surface and is crucial for efficient cellular
uptake of the protein to the lysosome.
Elosulfase alfa has a specific activity of 2.5 to 6.0 units/mg. One
activity unit is defined as the amount
of
the
enzyme
required
to
convert
1
micromole
of
sulfated
monosaccharide
substrate
D-
galactopyranoside-6-sulfate (Gal-6S) to de-sulfated-galactose (Gal)
and free sulfate per minute at
37°C.
Excipient(s) with known effect
Each 5 mL vial also contains 8 mg sodium and 100 mg sorbitol.
For the full list of excipients, see section 6.1.
3. PHARMACEUTICAL FORM
Concentrated solution for injection. A sterile, nonpyrogenic,
colourless to pale yellow and clear to
slightly opalescent solution with a pH between 5.0 to 5.8 that must be
diluted with sodium chloride 9
mg/mL (0.9%) solution for injection prior to administration.
4. CLINICAL PARTICULARS
4.1 THERAPEUTIC INDICATIONS
Vimizim is indicated for the treatment of mucopolysaccharidosis type
IVA (