Individual results may vary based on several factors, including severity of disease, initiation of treatment, and duration of therapy.
WHY SPINRAZA/LATER-ONSET STUDIES
The effectiveness of SPINRAZA has been studied in the longest clinical trial program in SMA to date.
Pivotal study:
CHERISH
CHERISH was a 15-month study in 126 people with later-onset SMA (Type 2 and Type 3), ages 2 to 9 years old. It was a randomized, sham-controlled study, which means individuals were randomly placed into a treated or untreated group.
The study evaluated the effect SPINRAZA has on motor function, including upper limb function.
The dosing schedule in the study was different than the approved SPINRAZA schedule.
Those treated with SPINRAZA experienced improvements over 15 months.
Those treated with SPINRAZA saw greater improvements on the HFMSE than the untreated group in just 3 months
RULM: The Revised Upper Limb Module assesses change in upper limb function through a series of tasks that reflect daily activities for both ambulatory and nonambulatory individuals with SMA; score ranges from 0 to 37 (if all activities are completed).
HFMSE: The Hammersmith Functional Motor Scale—Expanded is the standard measure used to assess motor function; includes skills such as lifting your head, sitting, and stair climbing. Each item is scored from 0 to 2, with a maximum score of 66.
Supportive studies:
CS2/CS12
CS2/CS12 was a 3-year study in 28 people with later-onset SMA (Type 2 and Type 3), ages 2 to 16 years old. The studies evaluated the safety and longer-term effects of SPINRAZA on upper limb and motor function, as well as walking ability, in later-onset SMA.
Limitations included: The dosing schedule was different than the approved SPINRAZA schedule, and the study was open-label, which means all people received SPINRAZA.
Type 2 individuals treated with SPINRAZA saw improvement from baseline in their upper limb function.
CHANGES IN UPPER LIMB FUNCTION
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.
Over the course of nearly 3 years (1150 days), 6 individuals with Type 2 SMA treated with SPINRAZA reached an average ULM score of 16.5, which is just 1.5 points below the maximum score of 18.
Individuals treated with SPINRAZA saw increases in motor function over 3 years.
CHANGES IN MOTOR FUNCTION
100% (7/7) of individuals with Type 3 SMA treated with SPINRAZA for at least 1050 days achieved meaningful improvements in their walking ability.
“For me, it’s always been mind over matter; focusing on what you can do, and working around what you can’t.”