Gene Therapy for Inherited Retinal Dystrophy

Coverage of subretinal voretigene neparvovec‑rzyl gene therapy for inherited retinal dystrophy due to biallelic RPE65 pathogenic or likely pathogenic variants in patients aged ≥12 months and <65 years with documented vision loss and viable retinal cells. Coverage requires genetic confirmation of biallelic RPE65 variants (homozygous or two variants in trans, with segregation testing if needed), meeting all policy and benefit‑plan criteria, and adherence to the labeled dosing (1.5×10^11 vector genomes in 0.3 mL per eye with separate administrations at least 6 days apart). Prior treatment of the intended eye, failure to meet age, genetic, or retinal‑viability requirements, or uses outside the approved indication are not covered.

"Requested therapy must be proven effective for the relevant diagnosis or procedure per generally accepted standards of practice."

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