No­var­tis an­nounces pos­i­tive da­ta from halt­ed Zol­gens­ma tri­al

The tri­al may be on hold, but No­var­tis says the drug works.

No­var­tis an­nounced to­day new da­ta on a de­liv­ery route to make the gene ther­a­py Zol­gens­ma ap­plic­a­ble and ac­ces­si­ble to old­er kids with spinal mus­cu­lar at­ro­phy, even as the Phase I/II tri­al on that route re­mains on hold. The FDA paused the tri­al in Oc­to­ber af­ter pre­clin­i­cal stud­ies showed loss of neur­al func­tion in some mice. The hold came af­ter an in­dus­try-rock­ing scan­dal over how No­var­tis han­dled da­ta-tam­per­ing in a dif­fer­ent set of pre­clin­i­cal Zol­gens­ma stud­ies.

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