Zolgensma

Country: Нови Зеланд

Језик: Енглески

Извор: Medsafe (Medicines Safety Authority)

Активни састојак:

Onasemnogene abeparvovec 20TVG/mL (TVG = Tera Vector Genomes; equivalent to 2 x 10^13 vector genomes per mL)

Доступно од:

Novartis New Zealand Ltd

Фармацеутски облик:

Solution for infusion

Састав:

Active: Onasemnogene abeparvovec 20TVG/mL (TVG = Tera Vector Genomes; equivalent to 2 x 10^13 vector genomes per mL) Excipient: Hydrochloric acid Magnesium chloride Poloxamer 188 Sodium chloride Trometamol Water for injection

Тип рецептора:

Prescription

Терапеутске индикације:

Zolgensma is an adeno-associated virus (AAV) vector-based gene therapy indicated for the treatment of paediatric patients with spinal muscular atrophy (SMA) with a bi allelic mutation in the survival motor neuron 1 (SMN1) gene.

Резиме производа:

Package - Contents - Shelf Life: Vial, plastic, Crystal zenith, cyclic olefin polymer vial closed with a chlorobutyl rubber stopper - 2 vials - 12 months from date of manufacture stored in the freezer at less than -60°C 14 days unopened stored at 2° to 8°C (Refrigerate, do not freeze) 8 hours opened stored in the infusion syringe - Vial, plastic, Crystal zenith, cyclic olefin polymer vial closed with a chlorobutyl rubber stopper - 3 vials - 12 months from date of manufacture stored in the freezer at less than -60°C 14 days unopened stored at 2° to 8°C (Refrigerate, do not freeze) 8 hours opened stored in the infusion syringe - Vial, plastic, Crystal zenith, cyclic olefin polymer vial closed with a chlorobutyl rubber stopper - 4 vials - 12 months from date of manufacture stored in the freezer at less than -60°C 14 days unopened stored at 2° to 8°C (Refrigerate, do not freeze) 8 hours opened stored in the infusion syringe - Vial, plastic, Crystal zenith, cyclic olefin polymer vial closed with a chlorobutyl rubber stopper - 5 vials - 12 months from date of manufacture stored in the freezer at less than -60°C 14 days unopened stored at 2° to 8°C (Refrigerate, do not freeze) 8 hours opened stored in the infusion syringe - Vial, plastic, Crystal zenith, cyclic olefin polymer vial closed with a chlorobutyl rubber stopper - 6 vials - 12 months from date of manufacture stored in the freezer at less than -60°C 14 days unopened stored at 2° to 8°C (Refrigerate, do not freeze) 8 hours opened stored in the infusion syringe - Vial, plastic, Crystal zenith, cyclic olefin polymer vial closed with a chlorobutyl rubber stopper - 7 vials - 12 months from date of manufacture stored in the freezer at less than -60°C 14 days unopened stored at 2° to 8°C (Refrigerate, do not freeze) 8 hours opened stored in the infusion syringe - Vial, plastic, Crystal zenith, cyclic olefin polymer vial closed with a chlorobutyl rubber stopper - 8 vials - 12 months from date of manufacture stored in the freezer at less than -60°C 14 days unopened stored at 2° to 8°C (Refrigerate, do not freeze) 8 hours opened stored in the infusion syringe - Vial, plastic, Crystal zenith, cyclic olefin polymer vial closed with a chlorobutyl rubber stopper - 9 vials - 12 months from date of manufacture stored in the freezer at less than -60°C 14 days unopened stored at 2° to 8°C (Refrigerate, do not freeze) 8 hours opened stored in the infusion syringe - Vial, plastic, Crystal zenith, cyclic olefin polymer vial closed with a chlorobutyl rubber stopper - 10 vials - 12 months from date of manufacture stored in the freezer at less than -60°C 14 days unopened stored at 2° to 8°C (Refrigerate, do not freeze) 8 hours opened stored in the infusion syringe - Vial, plastic, Crystal zenith, cyclic olefin polymer vial closed with a chlorobutyl rubber stopper - 11 vials - 12 months from date of manufacture stored in the freezer at less than -60°C 14 days unopened stored at 2° to 8°C (Refrigerate, do not freeze) 8 hours opened stored in the infusion syringe - Vial, plastic, Crystal zenith, cyclic olefin polymer vial closed with a chlorobutyl rubber stopper - 12 vials - 12 months from date of manufacture stored in the freezer at less than -60°C 14 days unopened stored at 2° to 8°C (Refrigerate, do not freeze) 8 hours opened stored in the infusion syringe - Vial, plastic, Crystal zenith, cyclic olefin polymer vial closed with a chlorobutyl rubber stopper - 13 vials - 12 months from date of manufacture stored in the freezer at less than -60°C 14 days unopened stored at 2° to 8°C (Refrigerate, do not freeze) 8 hours opened stored in the infusion syringe - Vial, plastic, Crystal zenith, cyclic olefin polymer vial closed with a chlorobutyl rubber stopper - 14 vials - 12 months from date of manufacture stored in the freezer at less than -60°C 14 days unopened stored at 2° to 8°C (Refrigerate, do not freeze) 8 hours opened stored in the infusion syringe

Датум одобрења:

2022-05-12

Карактеристике производа

NEW ZEALAND DATA SHEET
1. PRODUCT NAME
ZOLGENSMA
_®_
2 x 10
13
vector genomes/mL injection for intravenous infusion_ _
2. QUALITATIVE AND QUANTITATIVE COMPOSITION
Onasemnogene abeparvovec is a gene therapy medicinal product that
expresses the human survival
motor neuron (SMN) protein. It is a non-replicating recombinant
adeno-associated virus serotype 9
(AAV9) based vector containing the cDNA of the human SMN gene under
the control of the
cytomegalovirus enhancer/chicken-β-actin-hybrid promoter.
Onasemnogene abeparvovec is produced in human embryonic kidney cells
by recombinant DNA
technology.
Each mL contains onasemnogene abeparvovec with a nominal concentration
of 2 × 10
13
vector
genomes (vg). Vials will contain an extractable volume of not less
than either 5.5 mL or 8.3 mL. The
total number of vials and combination of fill volumes in each finished
pack will be customised to meet
dosing requirements for individual patients depending on their weight
(see sections 4.2 Dose and
method of administration and 6.5 Nature and contents of container).
Excipient with known effect
Zolgensma contains 0.2 mmol sodium per mL.
For the full list of excipients, see section 6.1
List of excipients.
3. PHARMACEUTICAL FORM
Injection for intravenous infusion.
When thawed, Zolgensma is a clear to slightly opaque, colorless to
faint white solution.
4. CLINICAL PARTICULARS
4.1 THERAPEUTIC INDICATIONS
Zolgensma is an adeno-associated virus (AAV) vector-based gene therapy
indicated for the
treatment of pediatric patients with spinal muscular atrophy (SMA)
with a bi-allelic mutation in the
survival motor neuron 1 (SMN1) gene.
4.2 DOSE AND METHOD OF ADMINISTRATION
Zolgensma should only be infused by a healthcare professional.
In order to improve the traceability of biological medicinal products,
the name and the batch
number of the administered product should be clearly recorded.
An immune response to the adeno-associated viral vector serotype 9
(AAV9) capsid will occur after
infusion of Zolgensma, thus patients should not be re-dosed w