WHY SPINRAZA/About SPINRAZA

SPINRAZA has been studied for 7+ years in the longest clinical trial program in SMA to date1

Studies have evaluated the effectiveness of SPINRAZA for improving survival, overall motor function, and walking ability1,2

TRIALS FOR LATER-ONSET SMA

Pivotal trial: CHERISH2,3

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 the Hammersmith Functional Motor Scale—Expanded (HFMSE)

Safety: The most common side effects were fever (43%), headache (29%), vomiting (29%), and back pain (25%)

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Supportive trial: CS2/CS124

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

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

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

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TRIALS FOR EARLY-ONSET AND PRESYMPTOMATIC SMA

Pivotal trial: ENDEAR2,5

Study: A phase 3, multicenter, double-blind, randomized (2:1), sham-controlled trial

Treatment duration: 13 months

Participants: 121 patients with early-onset SMA aged ≤7 months at time of first dose

Primary endpoints:

  • Improvement in motor function as measured by proportion of patients meeting the criteria for motor milestone responder using Hammersmith Infant Neurological Examination Section 2 (HINE-2)
  • Survival without the need for permanent ventilation

Safety: The most common side effects were lower respiratory infection (55%) and constipation (35%). Serious adverse reactions of atelectasis were more frequent in SPINRAZA-treated patients (18%) than in control patients (10%)

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Supportive trial: NURTURE6

Study: A phase 2, open-label, multicenter, multinational, single-arm trial

Treatment duration: Ongoing, results from a median of 2.9-year interim analysis

Participants: 25 presymptomatic, genetically diagnosed infants who were likely to develop Type 1 or 2 SMA aged ≤6 weeks at time of first dose

Primary endpoint: Survival without the need for permanent ventilation

Safety: Consistent with the SPINRAZA prescribing information

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