Roche oral therapy hits endpoint in older SMA patients, offering advantages over Zolgensma, Spinraza

After reporting positive data earlier this year in infants with spinal muscular atrophy, Roche’s risdiplam has now met the primary endpoint in a separate pivotal trial in older patients, paving the way for the therapy to reach a broader patient population than Zolgensma and compete with Spinraza on the basis of dosing convenience.

Zolgensma onasemnogene abeparvovec-xioi, a gene therapy from Novartis AG (NYSE:NVS; SIX:NOVN), is approved in SMA patients under two years of age. Spinraza nusinersen from Biogen Inc. (NASDAQ:BIIB) is approved in pediatric and adult patients. ...