Spark Therapeutics Announces Positive CHMP Opinion for One-time Gene Therapy LUXTURNA (voretigene neparvovec) in the European Union - Seite 2

A marketing authorization decision from the European Commission is anticipated approximately within two months. If approved, the authorization will be valid in all 28-member states of the European Union, as well as Iceland, Liechtenstein and Norway. In January 2018, Spark Therapeutics entered into a licensing and supply agreement with Novartis to commercialize LUXTURNA when and if approved in Europe and all markets outside the U.S.

LUXTURNA (voretigene neparvovec-rzyl) was approved by the U.S. Food & Drug Administration (FDA) in December 2017.

Clinical Trial Overview of LUXTURNA (voretigene neparvovec)
The safety and efficacy of LUXTURNA were assessed in one open-label, dose-exploration Phase 1 safety study (n=12), a second open-label Phase 1 safety study to assess the safety of injection of the contralateral eye (n=11) and an open-label, randomized, controlled Phase 3 efficacy and safety study (n=31) in pediatric and adult participants (range 4 to 44 years) with biallelic RPE65 mutation-associated retinal disease and sufficient viable retinal cells.

Of the 31 participants enrolled in the Phase 3 study, 21 were randomized to receive subretinal injection of LUXTURNA and 10 were randomized to the control (non-intervention) group. One participant in the intervention group discontinued from the study prior to treatment and one participant in the control group withdrew consent and was discontinued from the study. All nine participants randomized to the control group elected to cross over to receive LUXTURNA after one year of observation. All participants in these studies continue to be followed for long-term safety and efficacy. LUXTURNA Phase 3 clinical trial data, including data from the intervention group of all randomized participants through the one-year time point, have been previously reported in The Lancet.

The efficacy of LUXTURNA in the Phase 3 study was established based on the binocular multi-luminance mobility test (MLMT) score change from baseline to one year. MLMT was designed to measure changes in functional vision as assessed by the ability of a participant to navigate a course accurately and at a reasonable pace at seven different levels of illumination, ranging from 400 lux (corresponding to a brightly lit office) to one lux (corresponding to a moonless summer night). Each light level was assigned a score ranging from zero to six, with a higher score indicating that a participant could pass MLMT at a lower light level. A score of negative one was assigned to participants who could not pass MLMT at a light level of 400 lux. MLMT score change was defined as the difference between the score at baseline and the score at one year with a positive score change indicating that a participant was able to complete MLMT at a lower light level. Additional clinical outcomes included white light full-field light sensitivity threshold (FST) testing and visual acuity, both averaged over both eyes. Secondary endpoints included white light full-field light sensitivity threshold (FST) testing averaged over both eyes, first assigned eye MLMT and visual acuity.