País: Unión Europea
Idioma: inglés
Fuente: EMA (European Medicines Agency)
voretigene neparvovec
Novartis Europharm Limited
S01XA27
voretigene neparvovec
Other ophthalmologicals
Leber Congenital Amaurosis; Retinitis Pigmentosa
Luxturna is indicated for the treatment of adult and paediatric patients with vision loss due to inherited retinal dystrophy caused by confirmed biallelic RPE65 mutations and who have sufficient viable retinal cells.
Revision: 7
Authorised
2018-11-22
1 ANNEX I SUMMARY OF PRODUCT CHARACTERISTICS 2 This medicinal product is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions. See section 4.8 for how to report adverse reactions. 1. NAME OF THE MEDICINAL PRODUCT Luxturna 5 × 10 12 vector genomes/mL concentrate and solvent for solution for injection 2. QUALITATIVE AND QUANTITATIVE COMPOSITION 2.1 GENERAL DESCRIPTION Voretigene neparvovec is a gene transfer vector that employs an adeno-associated viral vector serotype 2 (AAV2) capsid as a delivery vehicle for the human retinal pigment epithelium 65 kDa protein (hRPE65) cDNA to the retina. Voretigene neparvovec is derived from wild-type AAV2 using recombinant DNA techniques. 2.2 QUALITATIVE AND QUANTITATIVE COMPOSITION Each mL of concentrate contains 5 × 10 12 vector genomes (vg). Each vial of Luxturna contains 0.5 extractable mL of concentrate (corresponding to 2.5 × 10 12 vector genomes) which requires a 1:10 dilution prior to administration, see section 6.6. After dilution of 0.3 mL of concentrate with 2.7 mL of solvent, each mL contains 5 × 10 11 vector genomes. Each dose of 0.3 mL Luxturna contains 1.5 x 10 11 vector genomes. For the full list of excipients, see section 6.1. 3. PHARMACEUTICAL FORM Concentrate and solvent for solution for injection. Following thaw from their frozen state, both the concentrate and the solvent are clear, colourless liquids with a pH of 7.3. 4. CLINICAL PARTICULARS 4.1 THERAPEUTIC INDICATIONS Luxturna is indicated for the treatment of adult and paediatric patients with vision loss due to inherited retinal dystrophy caused by confirmed biallelic _RPE65_ mutations and who have sufficient viable retinal cells. 3 4.2 POSOLOGY AND METHOD OF ADMINISTRATION Treatment should be initiated and administered by a retinal surgeon experienced in performing macular surgery. Posology Patients will receive a single dose of 1.5 × 10 11 vector genomes voretigene neparvovec i Leer el documento completo
1 ANNEX I SUMMARY OF PRODUCT CHARACTERISTICS 2 This medicinal product is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions. See section 4.8 for how to report adverse reactions. 1. NAME OF THE MEDICINAL PRODUCT Luxturna 5 × 10 12 vector genomes/mL concentrate and solvent for solution for injection 2. QUALITATIVE AND QUANTITATIVE COMPOSITION 2.1 GENERAL DESCRIPTION Voretigene neparvovec is a gene transfer vector that employs an adeno-associated viral vector serotype 2 (AAV2) capsid as a delivery vehicle for the human retinal pigment epithelium 65 kDa protein (hRPE65) cDNA to the retina. Voretigene neparvovec is derived from wild-type AAV2 using recombinant DNA techniques. 2.2 QUALITATIVE AND QUANTITATIVE COMPOSITION Each mL of concentrate contains 5 × 10 12 vector genomes (vg). Each vial of Luxturna contains 0.5 extractable mL of concentrate (corresponding to 2.5 × 10 12 vector genomes) which requires a 1:10 dilution prior to administration, see section 6.6. After dilution of 0.3 mL of concentrate with 2.7 mL of solvent, each mL contains 5 × 10 11 vector genomes. Each dose of 0.3 mL Luxturna contains 1.5 x 10 11 vector genomes. For the full list of excipients, see section 6.1. 3. PHARMACEUTICAL FORM Concentrate and solvent for solution for injection. Following thaw from their frozen state, both the concentrate and the solvent are clear, colourless liquids with a pH of 7.3. 4. CLINICAL PARTICULARS 4.1 THERAPEUTIC INDICATIONS Luxturna is indicated for the treatment of adult and paediatric patients with vision loss due to inherited retinal dystrophy caused by confirmed biallelic _RPE65_ mutations and who have sufficient viable retinal cells. 3 4.2 POSOLOGY AND METHOD OF ADMINISTRATION Treatment should be initiated and administered by a retinal surgeon experienced in performing macular surgery. Posology Patients will receive a single dose of 1.5 × 10 11 vector genomes voretigene neparvovec i Leer el documento completo