FDA lifts year-plus hold on No­var­tis' Zol­gens­ma, set­ting the stage for new PhI­II

Al­most two years af­ter the FDA or­dered an abrupt halt to No­var­tis’ test­ing of in­trathe­cal de­liv­ery of Zol­gens­ma, tri­als may fi­nal­ly re­sume.

The Swiss drug­mak­er says da­ta from its non-hu­man pri­mate tox­i­col­o­gy study have re­solved all safe­ty con­cerns reg­u­la­tors had, in­clud­ing any po­ten­tial risks of dor­sal root gan­glia, about this route of ad­min­is­tra­tion. No­var­tis’ next step will be ini­ti­at­ing STEER, a piv­otal Phase III study in SMA type 2 that will en­roll “treat­ment naïve pa­tients who are be­tween two and 18 years of age, able to sit, but have nev­er walked.”

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