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Spark Sets Sights On Bringing First Gene Therapy To Market In 2018

 
• By Jessica Merrill

A BLA for a potential gene therapy to correct vision loss due to certain inherited retinal disease has been accepted by FDA with a Jan. 12 PDUFA date. The proposed trade name is Luxturna.

Eye

Spark Therapeutics Inc. could bring the first gene therapy to the US market in 2018, after the company said it filed a biologic license application (BLA) for the drug has been accepted by the FDA for priority review with an action date of Jan. 12. The launch of a potential one-time treatment that could represent a cure for vision loss due to a certain inherited retinal disease (IRD) will pose new questions for the industry related to cost, reimbursement and the value of medicines.

The company announced July 17 that FDA has accepted its BLA for the gene therapy, voretigene neparvovec, with the proposed trade name of Luxturna. The treatment has the potential to be the first therapy for vision loss due to biallelic RPE65-mediated IRD

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