FDA New Drug and Biologic Approvals -- 2017 Year-in-Review

Mary L Windle, PharmD


January 11, 2018

In This Article

Luxturna (voretigene neparvovec-rzyl)


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.


Comments on Medscape are moderated and should be professional in tone and on topic. You must declare any conflicts of interest related to your comments and responses. Please see our Commenting Guide for further information. We reserve the right to remove posts at our sole discretion.