LUXTURNA- voretigene neparvovec-rzyl kit

מדינה: ארצות הברית

שפה: אנגלית

מקור: NLM (National Library of Medicine)

מרכיב פעיל:

VORETIGENE NEPARVOVEC (UNII: 2SPI046IKD) (VORETIGENE NEPARVOVEC - UNII:2SPI046IKD)

זמין מ:

Spark Therapeutics, Inc.

INN (שם בינלאומי):

VORETIGENE NEPARVOVEC

הרכב:

VORETIGENE NEPARVOVEC 0.05 mg

סוג מרשם:

PRESCRIPTION DRUG

סממני תרפויטית:

LUXTURNA (voretigene neparvovec-rzyl) is an adeno-associated virus vector-based gene therapy indicated for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy. Patients must have viable retinal cells as determined by the treating physician(s). None. Risk Summary Adequate and well-controlled studies with LUXTURNA have not been conducted in pregnant women. Animal reproductive studies have not been conducted with LUXTURNA. In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2-4% and 15-20%, respectively. Risk Summary There is no information regarding the presence of LUXTURNA in human milk, the effects on the breastfed infant, or the effects on milk production. The developmental and health benefits of breastfeeding should be considered along with the mother's clinical need for LUXTURNA and any potential adverse effects on the breastfed infant from LUXTURNA. No nonclinical or clinical studies were performed to evaluate the effect of LUXTURNA on fertility. Treatment with LUXTURNA is not recommended for patients younger than 12 months of age, because the retinal cells are still undergoing cell proliferation, and LUXTURNA would potentially be diluted or lost during cell proliferation. The safety and efficacy of LUXTURNA have been established in pediatric patients. Use of LUXTURNA is supported by Study 1 and Study 2 [see Clinical Studies (14) ] that included 25 pediatric patients with biallelic RPE65 mutation-associated retinal dystrophy in the following age groups: 21 children (age 4 years to less than 12 years) and 4 adolescents (age 12 years to less than 17 years). There were no significant differences in safety between the different age subgroups. The safety and effectiveness of LUXTURNA have not been established in geriatric patients. Clinical studies of LUXTURNA for this indication did not include patients age 65 years and over.

leaflet_short:

Each carton of LUXTURNA (NDC 71394 – 415-01) contains one single-dose vial of the LUXTURNA (NDC 71394 – 065-01, 0.5 mL extractable volume) and two vials of Diluent (NDC 71394 – 716-01, 1.7 mL extractable volume in each vial). LUXTURNA contains 5 x 1012 vector genomes (vg) per mL and requires a 1:10 dilution prior to administration. Store LUXTURNA and Diluent frozen at ≤ -65 °C. Following thaw of the vials, store at room temperature. Store diluted LUXTURNA at room temperature [See Dosage and Administration 2.2]. LUXTURNA is an adeno-associated virus vector-based gene therapy. Follow universal biohazard precautions for handling.

מצב אישור:

Biologic Licensing Application

מאפייני מוצר

LUXTURNA- VORETIGENE NEPARVOVEC-RZYL
SPARK THERAPEUTICS, INC.
----------
HIGHLIGHTS OF PRESCRIBING INFORMATION
THESE HIGHLIGHTS DO NOT INCLUDE ALL THE INFORMATION NEEDED TO USE
LUXTURNA SAFELY AND
EFFECTIVELY. SEE FULL PRESCRIBING INFORMATION FOR LUXTURNA.
LUXTURNA (VORETIGENE NEPARVOVEC-RZYL) INTRAOCULAR SUSPENSION FOR
SUBRETINAL INJECTION
INITIAL U.S. APPROVAL: 2017
INDICATIONS AND USAGE
LUXTURNA is an adeno-associated virus vector-based gene therapy
indicated for the treatment of patients
with confirmed biallelic _RPE65_ mutation-associated retinal
dystrophy. Patients must have viable retinal
cells as determined by the treating physician(s). (1)
DOSAGE AND ADMINISTRATION
FOR SUBRETINAL INJECTION ONLY.
The recommended dose of LUXTURNA for each eye is 1.5 x 10
vector genomes (vg), administered by
subretinal injection in a total volume of 0.3 mL. (2.1)
Perform subretinal administration of LUXTURNA to each eye on separate
days within a close interval,
but no fewer than 6 days apart. (2.1)
Recommend systemic oral corticosteroids equivalent to prednisone at 1
mg/kg/day (maximum of 40
mg/day) for a total of 7 days (starting 3 days before administration
of LUXTURNA to each eye), and
followed by a tapering dose during the next 10 days. (2.1)
DOSAGE FORMS AND STRENGTHS
LUXTURNA is a suspension for subretinal injection, supplied in a 0.5
mL extractable volume in a single-
dose 2 mL vial for a single administration in one eye. The supplied
concentration (5 x 10
vg/mL) requires
a 1:10 dilution prior to administration. The Diluent is supplied in
two single-use 2-mL vials. (3)
CONTRAINDICATIONS
None. (4)
WARNINGS AND PRECAUTIONS
Endophthalmitis: Use proper aseptic injection technique and monitor
for signs and symptoms of
infection. (5.1)
Permanent decline in visual acuity: Monitor for visual disturbances.
(5.2)
Retinal abnormalities: Monitor for macular abnormalities, retinal
tears or breaks and chorioretinal
atrophy. Do not inject in the immediate vicinity of the fovea. (5.3)
Increased intraocular pressure: Monitor and mana