CEO Jeff Marrazzo <\/p>\n
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. <\/p>\n
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. <\/p>\n
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. <\/p>\n
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 Gileads debut of its hep C cures, payers have become expert at establishing new rules limiting access. <\/p>\n
Thats what Spark wants to avoid. <\/p>\n
First, though, Spark has to win the FDAs OK, which will likely come with careful scrutiny of the data as well as the developing science of gene therapies. <\/p>\n
FDA acceptance for filing of our BLA for Luxturna is an important development for people living withRPE65-mediated IRD, a significant milestone for the gene therapy field, and a strong testament to the dedication of our collaborators and employees, saidMarrazzo in a statement. As we work closely withFDAin the months ahead, we will remain steadfast in our commitment to bring this important investigational therapy to people living withRPE65-mediated IRD who currently have no pharmacologic treatment options. <\/p>\n
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FDA embraces the first US application for a gene therapy, offering an accelerated test case - Endpoints News<\/a><\/p>\n","protected":false},"excerpt":{"rendered":" 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. Continue reading