Study: A phase 3, multicenter, randomized (2:1), double-blind, sham procedure–controlled trial
Treatment duration: 15 months
Participants: 126 patients with later-onset SMA, aged 2 to 9 years at screening
Primary endpoint: Change in motor function as measured by HFMSE
Secondary endpoint: Clinically meaningful change in HFMSE ≥3 points and change in upper limb function as measured by RULM
Study limitations: Differences in dosing compared to the approved SPINRAZA schedule
Safety: The most common side effects were fever (43%), headache (29%), vomiting (29%), and back pain (25%)
Study design: An independent, prospective, multicenter, observational cohort study
Study duration: Up to 14 months. Assessments made at 6, 10, and 14 months
Participants: 139 patients with genetically confirmed 5q later-onset SMA, aged 16 to 65
Primary endpoint: Change from baseline in motor function measured by HFMSE at 6, 10, and 14 months. Patients with missing baseline HFMSE scores were excluded from these analyses
Secondary endpoints: Change from baseline in upper limb and walking ability measured by RULM and 6MWT at 6, 10, and 14 months
Study limitations: No control group; observational design. Study powered on primary endpoint only. Statistics for other endpoints are descriptive only
Safety: The majority of adverse events (AEs) were generally consistent with those reported in the SPINRAZA clinical trials. Other reported AEs were:
Not a Biogen-sponsored study. Biogen-sponsored pivotal trials for SPINRAZA did not
include adults with SMA.
Study design: An independent, retrospective, multicenter, observational cohort study
Study duration: Up to 14 months. Assessments made at 6, 10, and 14 months
Participants: 116 patients with later-onset SMA Type 2 (n=13) and Type 3 (n=103), aged 18-72
Primary outcomes: Change from baseline in motor function measured by HFMSE, RULM, and 6MWT at 6, 10, and 14 months
Study limitations: No control group; retrospective observational design; missing data for some clinical assessment variables; and a small number of patients with SMA Type 2 (n=13)
Safety: The majority of adverse events (AEs) were generally consistent with those in the SPINRAZA clinial trials
Not a Biogen-sponsored study. Biogen-sponsored pivotal trials for SPINRAZA did not
include adults with SMA.
Study design: A real-world, independent critical review and meta-analysis assessing the efficacy of SPINRAZA in Type 2 and Type 3 SMA patients based on 19 peer-reviewed publications:
Motor outcomes measured: Hammersmith Functional Motor Scale—Expanded (HFMSE), Revised Upper Limb Module (RULM), and 6-Minute Walk Test (6MWT)
Study limitations:
Safety: Safety was not critically evaluated in this publication. Adverse events (AEs) reported in individual studies are included in the publication’s Supplemental Information section. AEs were generally consistent with those seen in the SPINRAZA pivotal trials5,9
Not a Biogen-sponsored study. Biogen-sponsored pivotal trials for SPINRAZA did not
include adults with SMA.