Positive two-year data for Evrysdi in infants with Type 1 SMA

New two-year data from Part 1 of the pivotal FIREFISH study of Evrysdi (risdiplam) in infants aged two-even months with symptomatic Type 1 spinal muscular atrophy (SMA) showed that they continued to improve and achieve motor milestones, according to Swiss pharma giant Roche (ROG: SIX), which leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics (Nasdaq: PTCT).

Analysts have previously forecast that risdiplam could bring in up to $2 billion at peak, partly due to its patient-friendly oral formulation, differentiating it from rival options in the SMA space, which are primarily gene therapies.

This exploratory analysis showed that an estimated 88% of infants were alive and required no permanent ventilation at two years. In addition, at two years, 59% (10/17 versus 7/17 at one year) of infants were able to sit without support for at least 5 seconds, assessed by the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development – Third Edition (BSID-III). 65% (11/17 vs 9/17 at 1-year) had maintained upright head control, 29% (5/17 vs 2/17 at one year) could turn themselves over and 30% (5/17 vs 1/17 at 1-year) were able to stand either supporting weight or with support.