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.

Unlock this article instantly by becoming a free subscriber.

You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.

SIGN UP LOG IN BECOME A PREMIUM SUBSCRIBER