Roche's risdiplam shows significant benefits for infants with Type 1 SMA

New one-year data from FIREFISH Part 2, a pivotal global study evaluating risdiplam in infants aged 1 – 7 months old with symptomatic Type 1 spinal muscular atrophy (SMA) showed positive results, according to Swiss pharma giant Roche (ROG: SIX).

The study met its primary endpoint with 29% of infants (12/41; p<0.0001) sitting without support for five seconds by month 12, as assessed by the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development Third Edition (BSID-III). No infants achieve this milestone in the natural history of Type 1 SMA.

On a day that Roche also released striking new data on its multiple sclerosis therapy Ocrevus (ocrelizumab), the company’s shares had gained 1.45% to 351.00 Swiss francs by late morning trading.