VIMIZIM elosulfase alfa (rch) 1 mg/mL concentrated solution for injection vial
Country: Australia
Language: English
Source: Department of Health (Therapeutic Goods Administration)
Patient Information leaflet
(PIL)
24-08-2020
Summary of Product characteristics
(SPC)
24-08-2020
Public Assessment Report
(PAR)
26-11-2017
Active ingredient:
elosulfase alfa, Quantity: 1 mg/mL
Available from:
BioMarin Pharmaceutical Australia Pty Ltd
INN (International Name):
elosulfase alfa
Pharmaceutical form:
Injection, concentrated
Composition:
Excipient Ingredients: arginine hydrochloride; monobasic sodium phosphate monohydrate; sodium acetate trihydrate; water for injections; polysorbate 20; sorbitol
Administration route:
Intravenous Infusion
Units in package:
1
Prescription type:
(S4) Prescription Only Medicine
Therapeutic indications:
Vimizim is indicated for the treatment of mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome).
Product summary:
Visual Identification: Colourless to pale yellow and clear to slightly opalescent solution.; Container Type: Vial; Container Material: Glass Type I Clear; Container Life Time: 36 Months; Container Temperature: Store at 2 to 8 degrees Celsius
Authorization status:
Licence status A
Authorization date:
2014-12-09
Patient Information leaflet
VIMIZIM
®
1
VIMIZIM
®
_elosulfase alfa (rch) _
CONSUMER MEDICINE INFORMATION
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 patients 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
REACTIONS WITH MEDICINES OR OTHER
ME
Read the complete document
Summary of Product characteristics
Product Information--Australia
V6c
Supersedes: V3
Page 1 of 16
AUSTRALIAN PI - VIMIZIM® (ELOSULFASE ALFA (RCH)) 1 MG/ML CONCENTRATED
SOLUTION FOR INJECTION
1. NAME OF THE MEDICINE
elosulfase alfa (rch)
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.
For the full list of excipients, see section 6.1 List of excipients.
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 (MPS IVA; Morquio A
syndrome).
4.2 DOSE AND METHOD OF ADMINISTRATION
Vimizim treatment should be supe
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