Usual Adult Dose for Retinal Disorder

1.5 x 10(11) vector genomes (vg) by subretinal injection into each eye on separate days

• Administration should be within a close interval, but separated by at least 6 days
• Total volume of injection should be 0.3 mL

• One dose of oral corticosteroid once a day for 7 days starting 3 days before procedure to first eye; follow with a dose taper during the next 10 days
• Dose: Prednisone 1 mg/kg/day or equivalent (maximum 40 mg/day)
• Second eye: Follow same regimen, but, if the taper following the first eye is not complete 3 days prior to scheduled surgery for the second eye, the corticosteroid regimen for the second eye should replace the taper for the first eye

• A short course of corticosteroids is recommended to limit the potential immune reaction.
• The treating physician must verify that the patient has viable retinal cells prior to the procedure.

Usual Pediatric Dose for Retinal Disorder

12 months or older: 1.5 x 10(11) vg by subretinal injection into each eye on separate days

• Administration should be within a close interval, but separated by at least 6 days
• Total volume of injection should be 0.3 mL

• One dose of oral corticosteroid once a day for 7 days starting 3 days before procedure to first eye; follow with a dose taper during the next 10 days
• Dose: Prednisone 1 mg/kg/day or equivalent (maximum 40 mg/day)
• Second eye: Follow same regimen, but, if the taper following the first eye is not complete 3 days prior to scheduled surgery for the second eye, the corticosteroid regimen for the second eye should replace the taper for the first eye.

• Treatment is not recommended for patients younger than 12 months because the retinal cells are still undergoing cell proliferation and this drug would potentially be diluted or lost during cell proliferation.
• A short course of corticosteroids is recommended to limit the potential immune reaction.
• The treating physician must verify that the patient has viable retinal cells prior to the procedure.

Renal Dose Adjustments

Data not available

Liver Dose Adjustments

Data not available

Precautions

CONTRAINDICATIONS: None

Safety and efficacy have not been established in patients older than 65 years.

Consult WARNINGS section for additional precautions.

Dialysis

Data not available

Other Comments

Administration advice:

• For subretinal injection only.
• A short course of corticosteroids starting 3 days prior to injection is recommended to limit a potential immune reaction.
• Therapy should be administered in a surgical suite under controlled aseptic conditions by a surgeon experienced in performing intraocular surgery.

• This drug and diluent should be stored frozen (minus 65C or lower).
• Thaw at room temperature prior to use.

• Requires a 1:10 dilution prior to administration
• Prepare injection within 4 hours of administration using sterile technique under aseptic conditions in a Class II vertical laminar flow biological safety cabinet
• See Manufacturer Prescribing Information for detailed preparation instructions.

• Administration will be once to each eye on separate days within a close interval, but not fewer than 6 days apart.
• This therapy is the first directly administered gene therapy approved in the US that targets a disease caused by mutation in a specific gene.

• Monitor for signs and symptoms of eye infection
• Monitor for macular abnormalities, retinal tears or breaks
• Monitor and manage intraocular pressure elevations
• Monitor for visual disturbances

• Patients should understand that serious infection, a decline in visual acuity, and retinal abnormalities may occur; patients should be instructed to notify their healthcare provider immediately if they experience new floaters, eye pain, flashes of light, or any changes in vision.
• Patients should be instructed to follow-up with their healthcare provider for measurement and management of intraocular pressure.
• Patients should be advised to avoid air travel, scuba diving, or travel to higher elevations until the air bubble formed with administration has completely dissipated from the eye; this may take a week or longer and they should receive clearance from their healthcare provider.
• Patients should understand that new or existing cataracts may get worse.
• Patients should be advised on proper handling of waste material generated from dressings, tears, and nasal secretions; handling precautions should be followed for up to 7 days.