No­var­tis touts sev­en years of dura­bil­i­ty da­ta for Zol­gens­ma

The same day that Roche tout­ed pos­i­tive dura­bil­i­ty and safe­ty da­ta for its spinal mus­cu­lar at­ro­phy drug Evrys­di, No­var­tis al­so made a splash with its mul­ti-mil­lion dol­lar gene ther­a­py for the dis­ease.

No­var­tis rolled out in­ter­im da­ta from two long-term fol­low-up stud­ies Mon­day at the 2023 Mus­cu­lar Dy­s­tro­phy As­so­ci­a­tion (MDA) Clin­i­cal and Sci­en­tif­ic Con­fer­ence. In the first study, LT-001, all chil­dren in the tri­al that were treat­ed af­ter show­ing symp­toms of SMA “main­tained all pre­vi­ous­ly achieved mo­tor mile­stones” up to 7.5 years af­ter be­ing dosed. The av­er­age time since Zol­gens­ma was giv­en was 6.86 years.

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