Voy­ager's shares sink af­ter ex­ecs say FDA has re­versed course on an ac­cel­er­at­ed fil­ing for its gene ther­a­py

The FDA ap­pears to have lost its ap­petite for an ac­cel­er­at­ed mar­ket­ing ap­pli­ca­tion that could pave the way to an ear­ly OK for Voy­ager’s lead gene ther­a­py. The com­pa­ny an­nounced af­ter the mar­ket closed Wednes­day that the agency’s view has shift­ed dra­mat­i­cal­ly. 

Af­ter tan­ta­liz­ing in­vestors in Ju­ly with its claim that FDA of­fi­cials said in a Type C ex­change that they were open to a fil­ing based on the safe­ty and ef­fi­ca­cy seen in a Phase II study for VY-AADC in Parkin­son’s dis­ease, the new word is that reg­u­la­tors are now view­ing the mid-stage study as a nec­es­sary pre­lim­i­nary step to a piv­otal tri­al. In an ad­den­dum that ar­rived in late Oc­to­ber, the biotech said, the FDA now says that “al­though the da­ta from the Phase II tri­al may sup­port the safe­ty and ef­fi­ca­cy of VY-AADC and could be con­sid­ered in the BLA re­view, the FDA cur­rent­ly con­sid­ers the Phase II tri­al as an ear­ly phase ex­plorato­ry study.”

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