Anthem Blue Cross Connecticut MED.00120 Gene Therapy for Ocular Conditions Form

This document addresses the use of gene therapy to treat inherited ophthalmic disease. Therapy can involve the supplementation of a defective gene or the introduction of a factor to decrease disease progression. Currently, only voretigene neparvovec-rzyl (Luxturna^®, Spark Therapeutics, Philadelphia, PA), a gene replacement therapy intended to treat retinal dystrophies caused by biallelic RPE65 gene mutations, has been approved by the FDA.

Note: Please see the following related document regarding genetic testing for individuals with inherited diseases, including inherited retinal diseases:

• CG-GENE-13 Genetic Testing for Inherited Diseases

Position Statement

Medically Necessary:

The use of voretigene neparvovec-rzyl is considered medically necessary in individuals who meet all of the following criteria:

1. A diagnosis of retinal dystrophy due to confirmed RPE65 mutation(s) in both alleles; and
2. At least 1 year of age; and
3. For each eye indicated for treatment, sufficient viable retinal cells as determined by non-invasive means, such as optical coherence tomography (OCT) and/or ophthalmoscopy as evidenced by one of the following:
   1. An area of retina within the posterior pole of greater than 100 µm thickness shown on OCT; or
   2. Three or more disc areas of retina without atrophy or pigmentary degeneration within the posterior pole; or
   3. Remaining visual field within 30 degrees of fixation as measured by a III4e isopter or equivalent;
      and
4. For each eye indicated for treatment, have not had intraocular surgery within 6 months.

Note: Voretigene neparvovec-rzyl has not been adequately studied or in individuals who are pregnant or unwilling to use contraception for 4 months after treatment.

Investigational and Not Medically Necessary:

The use of voretigene neparvovec-rzyl is considered investigational and not medically necessary when the above criteria are not met.

Repeat injections of voretigene neparvovec-rzyl in the same eye are considered investigational and not medically necessary in all cases.

The use of all other gene replacement therapies to treat any ocular condition is considered investigational and not medically necessary.