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Pharmaceuticals / Inspections and Audits

FDA Gives Priority Review Voucher for Retinal Dystrophy Drug

Jan. 5, 2018

The FDA issued a priority review voucher to Spark Therapeutics for its drug candidate Luxturna.

The drug is a gene therapy indicated for treatment of pediatric patients with biallelic RPE65 mutation-associated retinal dystrophy. Patients’ treating physicians must determine that they have viable retinal cells.

As an orphan drug, the candidate is exempt from requirements under the Pediatric Research Equity Act that it include an assessment of safety and effectiveness for pediatric patients in its application.

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