Roche Makes Case For Its Oral SMA Drug Risdiplam As Filings Beckon

Novartis may be turning up the rhetoric in spinal muscular atrophy as its cutting-edge gene therapy Zolgensma approaches a market currently enjoyed solely by Biogen’s Spinraza, but Roche is upbeat about the potential of its investigational oral product, risdiplam, to weigh in.

Speech
SMA companies talk up their products while data await • Source: Shutterstock

Roche is aiming for mid-year filings of its oral therapy for spinal muscular atrophy, risdiplam, based on promising Phase II/III data and positive regulator feedback. With a hoped-for approval in 2020, the product could add a convenient new therapy option into the SMA mix, and one that is likely to have its own impact on the treatment landscape.

SMA is the leading cause of genetic death in infants, affecting around one in 10,000 live births. The arrival of Biogen Inc

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