FDA ex­perts of­fer a unan­i­mous en­dorse­ment for Spark’s pi­o­neer­ing AAV gene ther­a­py for blind­ness

A pi­o­neer­ing AAV gene ther­a­py from Spark Ther­a­peu­tics $ONCE took a gi­ant stride to­ward an FDA ap­proval to­day as an out­side pan­el of ex­perts of­fered their sup­port for get­ting this game-chang­ing treat­ment in­to the mar­ket af­ter look­ing over the da­ta and hear­ing from some of the se­vere­ly sight-im­paired pa­tients whose lives had been trans­formed by this ther­a­py.

The vote was 16 to o fa­vor­ing the ben­e­fit-risk pro­file of the drug, back­ing an OK for voreti­gene nepar­vovec by the agency’s Cel­lu­lar, Tis­sue and Gene Ther­a­pies Ad­vi­so­ry Com­mit­tee and pro­vid­ing a com­pelling rea­son for the FDA to fol­low through with an his­toric first US ap­proval of a vec­tor-de­liv­ered gene ther­a­py.

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