Spark Therapeutics announced that the Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to its investigational product, SPK-RPE65 for the treatment of nyctalopis, or night blindness in patients with Leber’s congenital amaurosis due to mutations in the RPE65 gene.

The Breakthrough Therapy designation is supported by results from two clinical trials which suggest that SPK-RPE65 enables patients to perform activities of daily living with greater independence than prior to treatment. It also has long-lasting effects in restoring functional vision. The clinical team also reported that following a single injection of SPK-RPE65 in one eye, children in the trials no longer depended on visual aids to carry out classroom activities.

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SPK-RPE65 is currently being studied in a Phase 3 clinical trial. Data from that trial is expected to be released in the second half of 2015.

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