Voretigene Neparvovec-rzyl
Generic Name: voretigene neparvovec-rzyl
Brand Names:
Luxturna
11 DESCRIPTION LUXTURNA (voretigene neparvovec-rzyl) is a suspension of an adeno-associated virus vector-based gene therapy for subretinal injection. LUXTURNA is a live, non-replicating adeno-associated virus serotype 2 which has been genetically modified to express the human RPE65 gene. LUXTURNA is derived from naturally occurring adeno-associated virus using recombinant DNA techniques.
Overview
11 DESCRIPTION LUXTURNA (voretigene neparvovec-rzyl) is a suspension of an adeno-associated virus vector-based gene therapy for subretinal injection. LUXTURNA is a live, non-replicating adeno-associated virus serotype 2 which has been genetically modified to express the human RPE65 gene. LUXTURNA is derived from naturally occurring adeno-associated virus using recombinant DNA techniques.
Uses
1 INDICATIONS AND USAGE 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). 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
2 DOSAGE AND ADMINISTRATION For subretinal injection only. The recommended dose of LUXTURNA for each eye is 1.5 x 10 11 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 ) For subretinal injection only. 2.1 Dose The recommended dose of LUXTURNA for each eye is 1.5 x 10 11 vector genomes (vg), administered by subretinal injection in a total volume of 0.3 mL.
Side Effects
6 ADVERSE REACTIONS The most common adverse reactions (incidence ≥ 5%) were conjunctival hyperemia, cataract, increased intraocular pressure, retinal tear, dellen (thinning of the corneal stroma), macular hole, subretinal deposits, eye inflammation, eye irritation, eye pain, and maculopathy (wrinkling on the surface of the macula). The most common adverse reactions (incidence ≥ 5%) in the clinical trials were conjunctival hyperemia, cataract, increased intraocular pressure, retinal tear, dellen (thinning of the corneal stroma), macular hole, subretinal deposits, eye inflammation, eye irritation, eye pain, and maculopathy (wrinkling on the surface of the macula). ( 6 ) To report SUSPECTED ADVERSE REACTIONS, contact Spark Therapeutics, Inc.
Warnings
5 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 manage intraocular pressure elevations. ( 5.4 ) Expansion of intraocular air bubbles: Air travel and/or scuba diving is not recommended until any intraocular air bubbles have been absorbed. ( 5.5 ) Cataract: Subretinal injection of LUXTURNA may result in cataract formation or increase in the rate of cataract progression. 4 CONTRAINDICATIONS None. None.
Pregnancy
8.1 Pregnancy 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.
Storage
16 HOW SUPPLIED/STORAGE AND HANDLING 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 10 12 vector genomes (vg) per mL and requires a 1:10 dilution prior to administration.
Frequently Asked Questions
What is Voretigene Neparvovec-rzyl used for?▼
1 INDICATIONS AND USAGE 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). 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 )
What are the side effects of Voretigene Neparvovec-rzyl?▼
6 ADVERSE REACTIONS The most common adverse reactions (incidence ≥ 5%) were conjunctival hyperemia, cataract, increased intraocular pressure, retinal tear, dellen (thinning of the corneal stroma), macular hole, subretinal deposits, eye inflammation, eye irritation, eye pain, and maculopathy (wrinkling on the surface of the macula). The most common adverse reactions (incidence ≥ 5%) in the clinical trials were conjunctival hyperemia, cataract, increased intraocular pressure, retinal tear, dellen (thinning of the corneal stroma), macular hole, subretinal deposits, eye inflammation, eye irritation, eye pain, and maculopathy (wrinkling on the surface of the macula). ( 6 ) To report SUSPECTED ADVERSE REACTIONS, contact Spark Therapeutics, Inc.
Can I take Voretigene Neparvovec-rzyl during pregnancy?▼
8.1 Pregnancy 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.
What are the important warnings for Voretigene Neparvovec-rzyl?▼
5 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 manage intraocular pressure elevations. ( 5.4 ) Expansion of intraocular air bubbles: Air travel and/or scuba diving is not recommended until any intraocular air bubbles have been absorbed. ( 5.5 ) Cataract: Subretinal injection of LUXTURNA may result in cataract formation or increase in the rate of cataract progression. 4 CONTRAINDICATIONS None. None.
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Medical Disclaimer
This drug information is for educational purposes only and should not replace professional medical advice. Drug information is sourced from the FDA National Drug Code Directory and Structured Product Labeling. Always consult with a healthcare provider before starting, stopping, or changing any medication.