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, double-blind, randomized (2:1), sham-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%)

SEE RESULTS

TRIALS FOR EARLY-ONSET AND PRESYMPTOMATIC SMA

Pivotal trial: ENDEAR2,4

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

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: NURTURE5,6

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

Treatment duration: Ongoing

Participants: 25 presymptomatic infants who were genetically diagnosed with SMA (2 SMN2 copies n=15; 3 SMN2 copies n=10) and were aged ≤6 weeks

Primary endpoint: Survival without the need for permanent ventilation

Safety: Consistent with the SPINRAZA Prescribing Information. Latest 5-year data cut shows well-established safety and efficacy

SEE RESULTS

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