How Evrysdi helped patients with later-onset SMA
SUNFISH is a 2-part, multicenter trial to investigate the efficacy,
safety, and tolerability of Evrysdi in children and adults with Type 2
or Type 3 SMA. Learn
more about the SUNFISH trial design.
Motor function
Evrysdi demonstrated efficacy in children and adults with varying levels of disease severity
Evrysdi met its primary endpoint with significantly greater change
in motor function score vs placebo at Month 12, as measured by
MFM-32 1
- 1.55-point difference (95% CI: 0.30, 2.81) between the means (P=0.0156) 1
- The MFM-32 is a 32-item scale designed to assess various motor functions in people with neuromuscular disorders. The scale measures motor function abilities that relate to important daily function 10,11
Important Safety Information
Avoid coadministration with drugs that are substrates of multidrug
and toxin extrusion (MATE) transporters. Based on animal data, Evrysdi
may cause fetal harm and may compromise male fertility. Avoid use in
patients with hepatic impairment.
Please see additional important Safety Information below and full
Prescribing Information.
*The least square (LS) mean difference for change
from baseline in MFM-32 score (95% CI).
†Based on the missing-data rule for MFM-32, 6 patients
(n=5 for Evrysdi; n=1 for placebo) were excluded from the
analysis.
‡The mixed-model repeated-measures
analysis included the change from baseline total score as the
dependent variable; independent variables included the baseline
total score, treatment group, time, treatment-by-time interaction,
and the randomization stratification variable of age group (2 to 5,
6 to 11, 12 to 17, and 18 to 25 years). The MFM-32 total score was
calculated according to the user manual as a percentage of the
maximum score for the scale (ie, sum of the 32 item scores divided
by 96 and multiplied by 100).
MFM-32=Motor Function Measure-32 Items.
Upper limb
Evrysdi helped improve upper limb functions measured by RULM, such as picking up, lifting, and raising objects
RULM includes 20 items that measure the ability to push, pull,
place, tear, open, raise, and lift objects, as well as hand, arm, and
reaching movements in children and adults with SMA 12
-
1.59-point difference (95% CI: 0.55, 2.62)
between the means (P=0.0469)
*The LS mean difference for change from baseline
in RULM score (95% CI).
†Based on the missing-data
rule for RULM, 3 patients (n=1 for Evrysdi; n=2 for placebo) were
excluded
from the analysis.
‡The
mixed-model repeated-measures analysis included the change from
baseline total score as the
dependent variable; independent
variables included the baseline total score, treatment group,
time,
treatment-by-time interaction, and the randomization
stratification variable of age group (2 to 5, 6
to 11, 12 to
17, and 18 to 25 years).
RULM=Revised Upper Limb Module.