WHY SPINRAZA/LATER-ONSET EFFICACY

Pivotal trial: CHERISH3,5

Study: Phase 3, multicenter, randomized (2:1), double-blind, sham procedure–controlled

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: 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%)

Individuals treated with SPINRAZA experienced improvements in motor and upper limb function over 15 months.3

Those with later-onset SMA treated with SPINRAZA experienced a 3.9 improvement in motor function

HFMSE=Hammersmith Functional Motor Scale—Expanded.

Motor function began to steadily improve in just 6 months compared to untreated group.3

those with later-onset SMA treated with SPINRAZA experienced a 4.2 improvement in upper limb function

RULM=The Revised Upper Limb Module evaluates limb function and strength in individuals with later-onset SMA who are unable to walk. It is scored from 0-37 points, with higher scores indicating better function.

Supportive trials: CS2/CS124

Study: Phase 1b/2a, open-label, multiple-dose, dose-escalation integrated analysis of CS2 and CS12 studies

Treatment duration: ~3 years (38 months)

Participants: 28 ambulatory and nonambulatory patients with SMA aged 2 to 16 years at time of first dose

Primary endpoint: Safety of SPINRAZA

Study limitations: Differences in dosing compared to the approved SPINRAZA schedule and no control group

Safety: Adverse reactions were similar to those reported in the pivotal trials

Individuals treated with SPINRAZA saw increases in upper limb and motor function over 3 years.4

HFMSE was developed specifically for SMA and is a widely used and well-validated scale for measuring clinical benefit in later-onset SMA.6,7

ULM=The Upper Limb Module evaluates limb function and strength in individuals with later-onset SMA who are unable to walk. It is scored from 0-18 points, with higher scores indicating better function.

100% (7/7) who achieved meaningful improvements in their walking distance maintained these milestones through
year 3.8

those with Type 3 later-onset SMA treated with SPINRAZA increased their walking distance by 396.7 meters

SPINRAZA may help patients gain or regain the ability to walk.4

  • At least 1 of 11 patients with Type 2 SMA gained the ability to walk for the first time
  • At least 2 of 4 patients with Type 3 SMA regained the ability to walk
Ian Quote

“My belief is that anyone can do anything. It doesn’t matter if you’re in a wheelchair, it doesn’t take much strength to make
a big impact.”

–Byron

Review the warnings and precautions including thrombocytopenia, coagulation abnormalities, and renal toxicity.