With SPINRAZA, some patients achieved milestones they would not be expected to achieve, such as:
Individual results may vary based on several factors, including severity of disease and duration of therapy.
In open-label studies treating patients for up to 3 years, some patients sustained improvements in milestones at ages when they would otherwise be lost.1,2
- At least 1 of 11 patients with Type 2 SMA gained the ability to walk independently2
- At least 2 of 4 patients with Type 3 SMA regained the ability to walk independently2
The efficacy of SPINRAZA was demonstrated in ENDEAR and supported by an open-label study in patients with later-onset (Type 2 and Type 3) spinal muscular atrophy who maintained and increased motor function.1
Change in Hammersmith Functional Motor Scale Expanded (HFMSE) score from baseline after 1050 days2
The primary objective of this study was to examine the safety and tolerability of SPINRAZA in patients with Type 2 or Type 3 SMA. Additional exploratory endpoints included HFMSE, ULM (nonambulatory patients), and 6MWT (ambulatory patients).
Limitations of this study include that it was a longitudinal roll-over, re-dose, open-label study of 28 patients who received their fi rst dose in CS2 and then transferred to the second phase, CS12. Altogether, the dosage (3 mg-12 mg in CS2) and administration schedule in these studies differed from the approved SPINRAZA regimen.2
In a natural history cohort of later-onset (Type 2 and Type 3) SMA, mean change was -0.5 points over 24 months (730 days) and -1.7 points over 36 months (1095 days).3
In an open-label study, some ambulatory patients with later-onset spinal muscular atrophy were able to increase their walking distance in the 6-Minute Walk Test (6MWT) with SPINRAZA.2
Change in 6MWT distance from baseline after 1050 days2
In a natural history cohort, there was no significant change in 6MWT at 12 months.4
Later-onset (Type 3) SMA
treated with SPINRAZA