Safety
toxicity, pneumonia, risdiplam, retinal toxicity
PNEUMONIA, EYE DISORDERS
The safety profile after 24 months of treatment was consistent with the safety results after 12 months of treatment (Table Initial observations in the double-blind period showed a slightly higher incidence of serious pneumonia in patients in the risdiplam arm (8%) compared with the placebo arm (2%). However, the incidence of serious pneumonia did not increase in those patients who switched to risdiplam at month 12 (0%) compared with the first year on placebo (2%). Furthermore, the incidence of pneumonia did not increase during the second year of risdiplam treatment (7%), compared with the first year of risdiplam treatment (8%).A review of laboratory parameters did not reveal any risdiplam-associated toxicity. There have been no clinically significant safety findings in patients reflective of potential risks previously identified from nonclinical toxicology studies (effects on epithelial tissues, retinal toxicity, or hematologic effects). AEs in the System Organ Class ‘eye disorders’ were not suggestive of risdiplam-induced effects.