Spark Therapeutics completes regulatory filing application for its experimental gene therapy medicine

Written by Linda Loyd, Philadelphia Inquirer 

Drug developer Spark Therapeutics said Thursday that it had completed its biologics license application submission with the Food and Drug Administration for its experimental gene therapy for patients with rare inherited blindness.

If approved by the FDA, Spark’s lead drug candidate, voretigene neparvovec, could be the first U.S. treatment that uses genes as medicine.

The Philadelphia biotech, spun out of Children’s Hospital of Philadelphia, said the FDA will have 60 days to determine whether the application is complete. If the FDA decides the treatment should be given “priority review,” that would set a six-month target review period, said company spokeswoman Monique da Silva. “The review period is still to be determined.”

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