No­var­tis gains pri­or­i­ty re­view for SMA gene ther­a­py as in­vestors grap­ple with stick­er shock

An FDA ap­proval for the world’s first $4 mil­lion gene ther­a­py could ar­rive in as soon as five months.

No­var­tis says that reg­u­la­tors have opened up the ex­press­way for AVXS-101, a one-time treat­ment for spinal mus­cu­lar at­ro­phy type 1 that’s sup­posed to cure the dev­as­tat­ing neu­ro­mus­cu­lar dis­ease.

Now dubbed Zol­gens­ma, the gene ther­a­py first en­tered No­var­tis’ port­fo­lio this April through the $8.7 bil­lion AveX­is buy­out and has since be­come one of CEO Vas Narasimhan’s fa­vorite drugs in late-stage de­vel­op­ment. It is de­signed to cure SMA type 1 — restor­ing mo­tor func­tion and pre­vent­ing fur­ther mus­cle de­gen­er­a­tion — by in­sert­ing the gene re­spon­si­ble for the ail­ment.

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