Roche's SMA chal­lenge to Bio­gen's Spin­raza fran­chise looms larg­er with piv­otal win

Roche has just land­ed a cru­cial ad­vance in scor­ing a come-from-be­hind win on the spinal mus­cu­lar at­ro­phy field, giv­ing No­var­tis and Bio­gen a run for their mon­ey.

The up­date was brief, but Roche said ris­diplam hit the pri­ma­ry end­point in the place­bo-con­trolled piv­otal SUN­FISH tri­al, meet­ing the thresh­old for change from base­line in the Mo­tor Func­tion Mea­sure 32 (MFM-32) scale af­ter one year of treat­ment. The re­sults, which is the sec­ond, con­fir­ma­to­ry por­tion of a two-part study, in­volved 180 pa­tients with type 2 or 3 spinal mus­cu­lar at­ro­phy be­tween 2 and 25 years old.

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