FDA embraces the first US application for a gene therapy, offering an accelerated test case

Endpoints News – Spark Therapeutics is getting a short cut at the FDA for its lead gene therapy program, winning a priority review and a January 12, 2018 deadline for what may well become the first gene therapy approved in the US.

Spark is shooting for an FDA OK of Luxturna, better known in the trade as voretigene neparvovec, a gene therapy for RPE65-mediated inherited retinal disease.

If the approval does come through, Spark will be in the lead in establishing a reimbursement model for a gene therapy in a market dominated by multiple payers. UniQure tried and failed in Europe with Glybera and now GSK is following up making slow progress with Strimvelis.

Read more at Endpoints News.

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