No­var­tis takes a hit as FDA or­ders a hold on Zol­gens­ma tri­als in wake of fresh safe­ty con­cerns

The FDA has or­dered No­var­tis to halt en­roll­ment and dos­ing in a set of tri­als for Zol­gens­ma, their gene ther­a­py for lethal cas­es of spinal mus­cu­lar at­ro­phy. And the lat­est set­back trig­gered a fren­zy of an­a­lyst notes as they sized up who among No­var­tis’ ri­vals in the field would ben­e­fit most.

The hold was im­ple­ment­ed af­ter No­var­tis’ sub­sidiary AveX­is — still op­er­at­ing un­der the glare of an FDA in­ves­ti­ga­tion in­to an ethics scan­dal — in­formed reg­u­la­tors about con­cerns raised by an an­i­mal study point­ing to “dor­sal root gan­glia (DRG) mononu­clear cell in­flam­ma­tion, some­times ac­com­pa­nied by neu­ronal cell body de­gen­er­a­tion or loss.”

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