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

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.

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