More SMA data at AAN suggest new therapies could displace Spinraza

Novartis continued to add on new data for its spinal muscular atrophy gene therapy Zolgensma at AAN while Roche and Genentech presented updated SMA data for their oral small molecule risdiplam. The dosing and potential efficacy advantages for these agents could overtake Biogen and Ionis' Spinraza, which is currently the only treatment option in this orphan indication.

At the American Academy of Neurology meeting in Philadelphia, Roche (SIX:ROG; OTCQX:RHHBY) and its Genentech Inc. unit presented updated data from part 1 of the Phase II/III FIREFISH trial of risdiplam. Among 17 SMA type 1 patients who received the dose selected for the confirmatory part 2 portion of the trial, 7 (41.2%) were able to sit without support for at least five seconds, 11 (64.7%) were able to sit with or without support, nine (52.9%) had upright head control after 12 months of treatment and one (5.9%) was able to stand by 12 months...