Phesgo and Tecentriq Performance and Approvals
Evrysdi: 4 year data reinforce strong efficacy and safety profile
Mean change from baseline
in MFM32 total score**
Ph II/III (SUNFISH) results in SMA
Motor function score
3
N
6
12
18
24
30
36
42
48
Visit (months)
Risdiplam (n)ll
Placebo (n)ll
115 113 113 112
107
103
85
100
101
98
59 57 58 58
AEs per 100PY*
900
800
700
600
500
400
Ph II/III (SUNFISH) safety in SMA
H
300
200
100
0
O to ≤12
AE rates
I
SUNFISH Part 1
SUNFISH Part 2
Roche
Evrysdi.
risdiplam
MDA 2023
60mg
29
29
Risdiplam* (Months 0-48)
Placebos (Months 0-12)
>12 to ≤24
>24 to ≤36
Time on treatment (months)
• Increase in motor function scores achieved in first year sustained after 4 years, compared to natural history data
>36 to ≤48
• Overall AE rate decreased over 48 months, with AES and SAES reflective of underlying disease; treatment adherence at 99% and no
treatment-related AEs leading to withdrawal or treatment discontinuation
•
Diverse study population aged 2 to 25 years incl. more advanced disease (e.g. >65% of patients with scoliosis) reflecting a real-world
population underserved in clinical trials
Source: Natural history cohort, Roche data on file; courtesy of Association Institut de Myology; first presented at MDA 2021. MFM32-32-item motor function measure; SMA-spinal muscular atrophy; PY-patient years; AE-adverse
event; SAE-serious adverse event; *+/- 95% Confidence interval *Baseline is the last measurement prior to the first dose of risdiplam or placebo. *Data cut-off: 6 Sep 2022. §Data cut-off: 6 Sep 2019.; Number of patients with valid
results = number of patients with an available total score (result) at respective time pointsView entire presentation