FDA to Review Spark’s Retinal Gene Therapy Candidate Oct. 12

October 10, 2017: By Joan McKenna

SparkSpark Therapeutics’ gene therapy candidate for inherited retinal disease will be reviewed Oct. 12 by the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee.

The panel will consider whether to recommend voretigene neparvovec, branded Luxturna, for the treatment of patients with vision loss due to retinal dystrophy caused by flaws in the RPE65 gene.

Spark says the gene mutation breaks the visual cycle of light activating photoreceptors, resulting in progressive vision loss and often near blindness.

The FDA has granted orphan drug, breakthrough therapy, and rare pediatric disease designations for voretigene neparvovec, a one-time treatment.

Spark estimates that disease affects an estimated 1 thousand to 3 thousand patients in the US.

Philadelphia-based Spark, founded in March 2013, now has a workforce of 300 industry professionals and has secured $1 billion in financing. It has a pipeline of eight investigational gene therapies. Luxturna is its lead candidate.

The panel meeting will run 8:30 to 5 p.m. Oct. 12 at the FDA’s White Oak Campus in Silver Spring, Maryland.

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