Still held down by clin­i­cal hold, No­var­tis' Zol­gens­ma falls fur­ther be­hind Bio­gen and Roche as FDA asks for a new piv­otal study

Last Oc­to­ber, the FDA slowed down No­var­tis’ quest to ex­tend its gene ther­a­py to old­er spinal mus­cu­lar at­ro­phy pa­tients by slap­ping a par­tial hold on in­trathe­cal ad­min­is­tra­tion. Al­most a year lat­er, the hold is still there, and reg­u­la­tors are adding an­oth­er hur­dle re­quired for reg­u­la­to­ry sub­mis­sion: a new piv­otal con­fir­ma­to­ry study.

The new re­quire­ment — which de­parts sig­nif­i­cant­ly from No­var­tis’ pri­or ex­pec­ta­tions — will like­ly stretch the path to reg­is­tra­tion be­yond 2021, when an­a­lysts were ex­pect­ing a BLA sub­mis­sion. That could mean more time for Bio­gen to reap Spin­raza rev­enues and Roche to ramp up sales of Evrys­di in the ab­sence of a ri­val.

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