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    FDA approval of retinal dystrophy drug launches era of ocular gene therapy


    Improvements in MLMT scores

    The original intervention arm and the crossover patients who received delayed intervention showed statistically significant improvements in MLMT score, white-light full-field sensitivity, and visual field at the first outcome assessment on day 30 post-injection. The treatment benefits were sustained throughout follow-up.

    At their last test, 69% of all subjects were able to pass the MLMT at the lowest light level. Light sensitivity improved by about 100-fold in the original intervention group and almost 500-fold in the delayed intervention group. Change in BCVA was not significantly different between the intervention and control groups.

    “Improvements were reported 30 days after intervention,” said Dr. Falabella. Voretigene has the potential to provide lifelong efficacy because RPE cells are post-mitotic and unlikely to proliferate. Results so far support the expectation for sustained benefit, but, of course, only time will tell.”

    In the Phase III trial, one ocular serious adverse event was reported in one eye, in which there was foveal thinning and a sustained reduction in BCVA. The most common ocular adverse events were transient-elevated intraocular pressure, cataracts, retinal tears, and retinal deposits. No deleterious immune responses were reported.

    “Because the delivery procedure involves fluid-air exchange, patients also experience a decrease in vision for a few days,” Dr. Falabella said. “As the rods begin to capture more light, there were reports of early photophobia, but that was transient. The recommended minimum six-day interval between fellow eye injections allows for recovery of vision between procedures and for assessment of any potential complications or adverse events.”


    Cheryl Guttman Krader
    Cheryl Guttman is a medical writer based in Deerfield, Ill.


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