Confirmed biallelic retinal pigment epithelial-65 (RPE65) mutation-associated retinal dystrophy in adults and children aged 1 year or older.
Gene replacement therapy for RPE65 cells. RPE65 is produced in the retinal pigment epithelial (RPE) cells and converts all-trans-retinol to 11-cis-retinol, which subsequently forms the chromophore, 11-cis-retinal, during the visual (retinoid) cycle.
Per eye: 1.5 x 1011 vector genomes (vg) by subretinal injection in a total volume of 0.3 mL.
Approval was based on phase 3 clinical trial data, including data from the intent-to-treat population of all randomized participants through the one-year time point. Results showed a statistically significant and clinically meaningful difference between intervention (n=21) and control participants (n=10) at 1 year for mean bilateral multi-luminance mobility testing (MLMT) change score (difference of 1.6; 95% CI, 0.72, 2.41; P=0.001). Two secondary endpoints also showed improvement with treatment: full-field light sensitivity threshold (FST) testing (P<0.001) and the mobility test change score for the first injected eye (P=0.001).
Russell S, et al. Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial. Lancet. 2017 Aug 26;390(10097):849-60.
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Cite this: Mary L Windle. FDA New Drug and Biologic Approvals -- 2017 Year-in-Review - Medscape - Jan 11, 2018.