WHY SPINRAZA/About SPINRAZA

In 2016, SPINRAZA was the first FDA-approved treatment for SMA. Now it is approved in more than 50 countries worldwide.1,2*

How SPINRAZA
works

The first and only FDA-approved antisense oligonucleotide indicated for SMA.²

SPINRAZA has a targeted mechanism of action (MOA) in the central nervous system.

6+ years of data from the longest clinical trial program in SMA to date1

Studies have evaluated the effectiveness of SPINRAZA for improving survival, overall motor function, and walking ability.1,2

TRIALS FOR LATER-ONSET SMA

Pivotal trial: CHERISH2,3

Study: Phase 3, multicenter, randomized (2:1), double-blind, sham procedure–controlled

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
Supportive trial: CS2/CS124

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

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

SEE RESULTS

TRIALS FOR EARLY-ONSET AND PRESYMPTOMATIC SMA

Pivotal trial: ENDEAR2,5

Study: Phase 3, multicenter, double-blind, randomized (2:1), sham-controlled

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%)

SEE RESULTS
Supportive trial: NURTURE6

Study: Phase 2, open-label, multicenter, multinational, single-arm study

Treatment duration: Ongoing, results from a 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

SEE RESULTS

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

*As of December 2019.