Eye Disease

July 24, 2017

Spark Therapeutics has published phase III clinical trial data of voretigene neparvovec for the treatment of patients with vision loss due to biallelic RPE65-mediated inherited retinal disease (IRD). In the randomized, controlled gene therapy trial, voretigene neparvovec improved functional vision, light sensitivity and visual field. Results showed a statistically significant and clinically meaningful difference between intervention (n=21) and control participants (n=10) at one year, per the clinical trial’s primary endpoint, mean bilateral multi-luminance mobility testing (MLMT) change score (difference of 1.6; 95% CI, 0.72, 2.41; p=0.0013). Participants maintained functional gains observed 30 days post-administration at the one-year primary endpoint. MLMT evaluates functional vision by documenting the participants’ ability to navigate a mobility course under a variety of specified light levels ranging from one lux (equivalent to, for example, a moonless summer night) to 400 lux (equivalent to, for example, an office environment). In May 2017, Spark Therapeutics completed the rolling submission of a BLA with the FDA.

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