Description

What Is Luxturna(luxturna price | luxturna cost)?

luxturna price | luxturna cost  (voretigene neparvovec-rzyl) is an adeno-associated virus vector-based gene therapy indicated for the treatment of patients with confirmed biallelic RPE65mutation-associated retinal dystrophy.

What Are Side Effects of luxturna price | luxturna cost?

luxturna price | luxturna cost may cause serious side effects including:

• hives,
• difficulty breathing,
• swelling of your face, lips, tongue, or throat,
• new or worsening cataract (clouding of the lens inside of the eye),
• thinning of the clear layer in front of your eye,
• deposits under your retina,
• separating layers of a developing hole in the center of your retina,
• thinning of the retina or loss of function,
• breaks or wrinkling on the surface of your retina,
• retinal detachment,
• bleeding in the retina,
• permanent decline in the sharpness of your vision,
• new or worsening vision problems,
• flashes of light or “floaters” in your vision,
• seeing halos around lights,
• eye pain or swelling,
• headache,
• increased sensitivity to light,
• crusting or drainage from the eye, and
• vision loss

Get medical help right away, if you have any of the symptoms listed above.

Common side effects include:

• eye redness,
• cataracts,
• 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)

Seek medical care or call 911 at once if you have the following serious side effects:

• Serious eye symptoms such as sudden vision loss, blurred vision, tunnel vision, eye pain or swelling, or seeing halos around lights;
• Serious heart symptoms such as fast, irregular, or pounding heartbeats; fluttering in your chest; shortness of breath; and sudden dizziness, lightheadedness, or passing out;
• Severe headache, confusion, slurred speech, arm or leg weakness, trouble walking, loss of coordination, feeling unsteady, very stiff muscles, high fever, profuse sweating, or tremors.

This document does not contain all possible side effects and others may occur. Check with your physician for additional information about side effects.

Dosage for luxturna price | luxturna cost

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. Perform subretinal administration of Luxturna to each eye on separate days within a close interval, but no fewer than 6 days apart.

What Drugs, Substances, or Supplements Interact with Luxturna?

Luxturna may interact with other drugs. Tell your doctor all medications and supplements you use.

luxturna price | luxturna cost During Pregnancy and Breastfeeding

Tell your doctor if you are pregnant or plan to become pregnant before using Luxturna; it is unknown if it would affect a fetus. It is unknown if Luxturna passes into breast milk. Consult your doctor before breastfeeding.

Additional Information

Our Luxturna (voretigene neparvovec-rzyl) Intraocular Suspension for Subretinal Injection Side Effects Drug Center provides a comprehensive view of available drug information on the potential side effects when taking this medication.

Uses for Luxturna (Voretigene neparvovec) | luxturna price | luxturna cost

Luxturna (Voretigene neparvovec) | luxturna price | luxturna cost (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).

Dosage for Luxturna (Voretigene neparvovec) | luxturna price | luxturna cost

For subretinal injection only.

Dose

• The recommended dose of Luxturna (Voretigene neparvovec) | luxturna price | luxturna cost for each eye is 1.5 x 10¹¹ vector genomes (vg), administered by subretinal injection in a total volume of 0.3 mL.
• Perform subretinal administration of Luxturna (Voretigene neparvovec) | luxturna price | luxturna cost to each eye on separate days within a close interval, but no fewer than 6 days apart.
• 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 (Voretigene neparvovec) | luxturna price | luxturna cost to the first eye), and followed by tapering the dose during the following 10 days. The same corticosteroid dosing regimen applies for the administration of Luxturna (Voretigene neparvovec) | luxturna price | luxturna cost to the second eye. If the corticosteroid taper following Luxturna (Voretigene neparvovec) | luxturna price | luxturna cost administration to the first eye is not complete three days prior to the planned LUXTURNA administration to the second eye, then the corticosteroid regimen for the second eye replaces the taper for the first eye.

Preparation

Prepare Luxturna (Voretigene neparvovec) | luxturna price | luxturna cost within 4 hours of administration using sterile technique under aseptic conditions in a Class II vertical laminar flow biological safety cabinet (BSC). Below is the list of items required for dilution and administration syringe preparation:

• One single-dose vial of Luxturna (Voretigene neparvovec) | luxturna price | luxturna cost
• Two vials of Diluent
• One 3-mL sterile syringe
• One 20G 1-inch sterile needle
• Three 1-mL sterile syringes
• Three 27G ½-inch sterile needles
• Two sterile syringe caps
• One 10-mL sterile empty glass vial
• One sterile utility drape
• One sterile plastic bag
• Two sterile labels for administration syringes
• One sterile plain label
• One sterile skin marker

The U.S. Food and Drug Administration today approved Luxturna (Voretigene neparvovec) | luxturna price | luxturna cost (voretigene neparvovec-rzyl), a new gene therapy, to treat children and adult patients with an inherited form of vision loss that may result in blindness. Luxturna is the first directly administered gene therapy approved in the U.S. that targets a disease caused by mutations in a specific gene.

“Today’s approval marks another first in the field of gene therapy — both in how the therapy works and in expanding the use of gene therapy beyond the treatment of cancer to the treatment of vision loss — and this milestone reinforces the potential of this breakthrough approach in treating a wide-range of challenging diseases.

The culmination of decades of research has resulted in three gene therapy approvals this year for patients with serious and rare diseases. I believe gene therapy will become a mainstay in treating, and maybe curing, many of our most devastating and intractable illnesses,” said FDA Commissioner Scott Gottlieb, M.D.

“We’re at a turning point when it comes to this novel form of therapy and at the FDA, we’re focused on establishing the right policy framework to capitalize on this scientific opening. Next year, we’ll begin issuing a suite of disease-specific guidance documents on the development of specific gene therapy products to lay out modern and more efficient parameters — including new clinical measures — for the evaluation and review of gene therapy for different high-priority diseases where the platform is being targeted.”

Luxturna (Voretigene neparvovec) | luxturna price | luxturna cost is approved for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy that leads to vision loss and may cause complete blindness in certain patients.

Hereditary retinal dystrophies are a broad group of genetic retinal disorders that are associated with progressive visual dysfunction and are caused by mutations in any one of more than 220 different genes. Biallelic RPE65 mutation-associated retinal dystrophy affects approximately 1,000 to 2,000 patients in the U.S. Biallelic mutation carriers have a mutation (not necessarily the same mutation) in both copies of a particular gene (a paternal and a maternal mutation).

The RPE65 gene provides instructions for making an enzyme (a protein that facilitates chemical reactions) that is essential for normal vision. Mutations in the RPE65 gene lead to reduced or absent levels of RPE65 activity, blocking the visual cycle and resulting in impaired vision. Individuals with biallelic RPE65 mutation-associated retinal dystrophy experience progressive deterioration of vision over time. This loss of vision, often during childhood or adolescence, ultimately progresses to complete blindness.

Luxturna (Voretigene neparvovec) | luxturna price | luxturna cost works by delivering a normal copy of the RPE65 gene directly to retinal cells. These retinal cells then produce the normal protein that converts light to an electrical signal in the retina to restore patient’s vision loss. Luxturna uses a naturally occurring adeno-associated virus, which has been modified using recombinant DNA techniques, as a vehicle to deliver the normal human RPE65 gene to the retinal cells to restore vision.

“The approval of Luxturna further opens the door to the potential of gene therapies,” said Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research (CBER). “Patients with biallelic RPE65 mutation-associated retinal dystrophy now have a chance for improved vision, where little hope previously existed.”

Luxturna (Voretigene neparvovec) | luxturna price | luxturna cost should be given only to patients who have viable retinal cells as determined by the treating physician(s). Treatment with Luxturna must be done separately in each eye on separate days, with at least six days between surgical procedures. It is administered via subretinal injection by a surgeon experienced in performing intraocular surgery. Patients should be treated with a short course of oral prednisone to limit the potential immune reaction to Luxturna.

The safety and efficacy of Luxturna (Voretigene neparvovec) | luxturna price | luxturna cost were established in a clinical development program with a total of 41 patients between the ages of 4 and 44 years. All participants had confirmed biallelic RPE65 mutations. The primary evidence of efficacy of Luxturna was based on a Phase 3 study with 31 participants by measuring the change from baseline to one year in a subject’s ability to navigate an obstacle course at various light levels.

The group of patients that received Luxturna demonstrated significant improvements in their ability to complete the obstacle course at low light levels as compared to the control group.

The most common adverse reactions from treatment with Luxturna included eye redness (conjunctival hyperemia), cataract, increased intraocular pressure and retinal tear.

The FDA granted this application Priority Review and Breakthrough Therapy designations. Luxturna also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.

The sponsor is receiving a Rare Pediatric Disease Priority Review Voucher under a program intended to encourage development of new drugs and biologics for the prevention and treatment of rare pediatric diseases. A voucher can be redeemed by a sponsor at a later date to receive Priority Review of a subsequent marketing application for a different product. This is the 13th rare pediatric disease priority review voucher issued by the FDA since the program began.

To further evaluate the long-term safety, the manufacturer plans to conduct a post-marketing observational study involving patients treated with Luxturna.

The FDA granted approval of Luxturna to Spark Therapeutics Inc.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines, and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

Related Information

• Luxturna (voretigene neparvovec-rzyl) product page
• Consumer Update: What Is Gene Therapy? How Does It Work?
• Cellular and Gene Therapy Products
• What is Gene Therapy?
• Approved Cellular and Gene Therapy Products

Luxturna is the first gene therapy approved in the U.S. to target a disease caused by mutations in a specific gene