Elelyso

Country: New Zealand

Language: English

Source: Medsafe (Medicines Safety Authority)

Active ingredient:

Taliglucerase alfa 200 U (200 U deliverable, plus 6% overage)

Available from:

Pfizer New Zealand Limited

INN (International Name):

Taliglucerase alfa 200 U (200 U deliverable, plus 6% overage)

Dosage:

200 U

Pharmaceutical form:

Powder for injection

Composition:

Active: Taliglucerase alfa 200 U (200 U deliverable, plus 6% overage) Excipient: Citric acid Mannitol Polysorbate 80 Sodium citrate dihydrate

Prescription type:

Prescription

Manufactured by:

Protalix Ltd.

Therapeutic indications:

Elelyso is indicated for long-term enzyme replacement therapy for adult and paediatric patients with a confirmed diagnosis of Type 1 Gaucher disease associated with at least one of the following: splenomegaly, hepatomegaly, anaemia, thrombocytopenia.

Product summary:

Package - Contents - Shelf Life: Vial, glass, - 1 dose units - 24 months from date of manufacture stored at 2° to 8°C (Refrigerate, do not freeze) protect from light 24 hours diluted stored at 2° to 8°C (Refrigerate, do not freeze). Reconstituted and diluted, total storage time not to exceed 24h

Authorization date:

2016-08-31

Patient Information leaflet

Elelyso®
1
ELELYSO®
CONSUMER MEDICINE INFORMATION (CMI) SUMMARY
The full CMI on the next page has more details. If you are worried
about using this medicine, speak to your doctor or pharmacist.
1.
WHY AM I USING ELELYSO?
Elelyso contains the active ingredient taliglucerase alfa rpc and is
an Enzyme Replacement Therapy which is used to treat Type 1
Gaucher disease in adults and children (2 to 17 years of age) with at
least one of the following signs of the condition: a spleen or
liver that is larger than expected; a low number of red blood cells
(anaemia); a tendency to bleed easily caused by a low blood
platelet count (platelets stop blood loss by forming a plug inside a
blood vessel). For more information, see Section 1. Why am I
using Elelyso? in the full CMI.
2.
WHAT SHOULD I KNOW BEFORE I USE ELELYSO?
Do not use if you have ever had an allergic reaction to any medicine
containing taliglucerase alfa rpc, other Enzyme Replacement
Therapies such as Cerezyme® or Vpriv®, to carrots, or any of the
ingredients listed at the end of the CMI.
Talk to your doctor if you have previously experienced a reaction
during an infusion or allergic reaction to other Enzyme
Replacement Therapy for Gaucher disease; if you are on a low salt
diet; if you have experienced difficulty breathing; if you take
any other medicines; or if you are pregnant or plan to become pregnant
or are breastfeeding.
For more information, see Section 2. What should I know before I use
Elelyso? in the full CMI.
3.
WHAT IF I AM TAKING OTHER MEDICINES?
Some medicines may interfere with Elelyso and affect how it works. For
more information, see Section 3. What if I am taking
other medicines? in the full CMI.
4.
HOW DO I USE ELELYSO?
Elelyso should only be given under the supervision of a doctor (or
healthcare professional) who is knowledgeable in the
treatment of Gaucher disease. Your doctor will decide the dosage and
the frequency of the dose suitable for you.
More instructions can be found in Section 4. How do I use Elelyso? in
the full CMI.
5.
WHAT SHO

Summary of Product characteristics

Version: pfdelelv10822
Supersedes: pfdelelv11119
Page 1 of 18
NEW ZEALAND DATA SHEET
1.
PRODUCT NAME
ELELYSO
®
(taliglucerase alfa rpc) 200 units powder for injection
2.
QUALITATIVE AND QUANTITATIVE COMPOSITION
Each vial contains 200 units* of taliglucerase alfa rpc**.
Taliglucerase alfa rpc is a glycosylated protein with approximately 7%
of its molecular mass
contributed
by
glycans.
The
glycans
present
in
taliglucerase
alfa
rpc
are
typical
of
plant-expressed proteins. The most abundant glycan has terminal
mannose, β-(1,2)-xylose, and
α-(1,3)-fucose residues.
The terminal mannose residues specifically bind the endocytic
mannose receptors on macrophages, resulting in uptake of the enzyme
into the macrophages,
the cells that accumulate lipid in Gaucher disease and are the target
cells for enzyme
replacement therapy.
The glycan structures β-(1,2)-xylose, and α-(1,3)-fucose residues
are
widely present in plant but not in mammalian glycoproteins.
Taliglucerase alfa rpc sequence contains seven cysteine residues that
form two disulfide bonds
between the first four cysteine residues (Cys6-Cys18, Cys20-Cys25) and
three free sulfhydryls
(thiols).
Predicted Amino Acid Sequence for taliglucerase alfa rpc:
1
11
21
31
41
51
1
EFARPCIPKS
FGYSSVVCVC
NATYCDSFDP
PTFPALGTFS
RYESTRSGRR
MELSMGPIQA
61
NHTGTGLLLT
LQPEQKFQKV
KGFGGAMTDA
AALNILALSP
PAQNLLLKSY
FSEEGIGYNI
121
IRVPMASCDF
SIRTYTYADT
PDDFQLHNFS
LPEEDTKLKI
PLIHRALQLA
QRPVSLLASP
181
WTSPTWLKTN
GAVNGKGSLK
GQPGDIYHQT
WARYFVKFLD
AYAEHKLQFW
AVTAENEPSA
241
GLLSGYPFQC
LGFTPEHQRD
FIARDLGPTL
ANSTHHNVRL
LMLDDQRLLL
PHWAKVVLTD
301
PEAAKYVHGI
AVHWYLDFLA
PAKATLGETH
RLFPNTMLFA
SEACVGSKFW
EQSVRLGSWD
361
RGMQYSHSII
TNLLYHVVGW
TDWNLALNPE
GGPNWVRNFV
DSPIIVDITK
DTFYKQPMFY
421
HLGHFSKFIP
EGSQRVGLVA
SQKNDLDAVA
LMHPDGSAVV
VVLNRSSKDV
PLTIKDPAVG
481
FLETISPGYS
IHTYLWHRQD
LLVDTM
After reconstitution, the solution contains 40 units (approximately
1.2 mg) of taliglucerase alfa
rpc per mL (200 units/5 mL).
* An enzyme unit is defined as the amount of enzyme that catalyses the
hydrolysis of one
micromo