Drug Development

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

CEO Jeff Marrazzo

Spark Therapeutics $ONCE 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.

But how will US payers react to a once-and-done therapy that could easily cost 7 figures? Spark CEO Jeff Marrazzo has been thinking on that for several years now, wondering how best to structure payments for a rare disease like this. Developers of all stripes have been offering concessions like money-back guarantees to win over payers for pricey new drugs. But since Gilead’s debut of its hep C cures, payers have become expert at establishing new rules limiting access.

That’s what Spark wants to avoid.

First, though, Spark has to win the FDA’s OK, which will likely come with careful scrutiny of the data as well as the developing science of gene therapies.

“FDA acceptance for filing of our BLA for Luxturna is an important development for people living with RPE65-mediated IRD, a significant milestone for the gene therapy field, and a strong testament to the dedication of our collaborators and employees,” said Marrazzo in a statement. “As we work closely with FDA in the months ahead, we will remain steadfast in our commitment to bring this important investigational therapy to people living with RPE65-mediated IRD who currently have no pharmacologic treatment options.”


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