WHY SPINRAZA/ABOUT SPINRAZA

SPINRAZA has been studied for over 8 years in the longest SMA clinical trial program to date1

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

TRIALS FOR PRESYMPTOMATIC AND EARLY-ONSET SMA

Supportive trial: NURTURE3,4

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

Treatment duration: Ongoing (latest data cut is after approximately 5 years of follow up)

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 respiratory intervention

Safety: Consistent with the SPINRAZA Prescribing Information

SEE RESULTS
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

Primary endpoints:

  • Improvement in motor function as measured by proportion of patients meeting the criteria for motor milestone responder using 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%)

HINE-2=Hammersmith Infant Neurological
Examination Section 2.

SEE RESULTS

TRIAL FOR LATER-ONSET SMA

Pivotal trial: CHERISH2,6

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

Treatment duration: 15 months

Participants: 126 patients with later-onset SMA aged 2 to 9 years at screening

Primary endpoint: Least-squares mean change from baseline in HFMSE at 15 months of treatment

Select secondary endpoints: Clinically meaningful change in HFMSE ≥3 points and 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%)

HFMSE=Hammersmith Functional Motor Scale—Expanded; RULM=Revised Upper Limb Module.

SEE RESULTS

Review the warnings and precautions, including thrombocytopenia, coagulation abnormalities, and renal toxicity2