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CHERISH primary endpoint: change in HFMSE

Some patients with later-onset spinal muscular atrophy treated with SPINRAZA achieved improvements in motor function.1

Change from baseline in HFMSE total score at month 151

The mean HFMSE score at baseline was 22.4 for SPINRAZA compared with 19.9 for untreated patients out of a maximum score of 66.1

HFMSE=Hammersmith Functional Motor Scale—Expanded; SE=standard error; LSM=least squares mean; a mathematical analysis that accounts for the estimation of missing data values of children who had not completed the study at the time of the efficacy analysis.1-3

Key Icon

Patients treated with SPINRAZA demonstrated a clinically meaningful change (≥3-point increase) in HFMSE total score from baseline, improving in ≥2 motor skills

SPINRAZA helps maintain motor function in open-label CS2/CS12 studies

In a longitudinal analysis of 2 open-label studies, ambulatory and nonambulatory patients with later-onset (Type 2 and Type 3) spinal muscular atrophy maintained and increased motor function.4

Change in HFMSE score from baseline after 1050 days4

12.3-point increase
from baseline of 21.3 at day 10504

1.6-point increase
from baseline of 48.9 at day 10504

Key Icon

In a natural history cohort of later-onset (Type 2 and Type 3) spinal muscular atrophy, mean change was -0.5 points over 24 months (730 days) and -1.7 points over 36 months (1095 days).5

image of Emma aged 7 years old with Type 3 SMA swimming
Emma
age 7

Later-onset (Type 3) SMA treated with SPINRAZA

Individual results may vary based on several factors, including severity of disease and duration of therapy.

INDICATION

SPINRAZA is indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.

IMPORTANT SAFETY INFORMATION

Coagulation abnormalities and thrombocytopenia, including acute severe thrombocytopenia, have been observed after administration of some antisense oligonucleotides. Patients may be at increased risk of bleeding complications.

In the sham-controlled studies for patients with infantile-onset and later-onset SMA, 24 of 146 SPINRAZA-treated patients (16%) with high, normal, or unknown platelet count at baseline developed a platelet level below the lower limit of normal, compared to 10 of 72 sham-controlled patients (14%). Two SPINRAZA-treated patients developed platelet counts <50,000 cells per microliter, with the lowest level of 10,000 cells per microliter recorded on study day 28.

Renal toxicity, including potentially fatal glomerulonephritis, has been observed after administration of some antisense oligonucleotides. SPINRAZA is present in and excreted by the kidney. In the sham-controlled studies for patients with infantile-onset and later-onset SMA, 71 of 123 SPINRAZA-treated patients (58%) had elevated urine protein, compared to 22 of 65 sham-controlled patients (34%).

Laboratory testing and monitoring to assess safety should be conducted. Perform a platelet count, coagulation laboratory testing, and quantitative spot urine protein testing at baseline and prior to each dose of SPINRAZA and as clinically needed.

Severe hyponatremia was reported in an infant treated with SPINRAZA requiring salt supplementation for 14 months.

Cases of rash were reported in patients treated with SPINRAZA.

SPINRAZA may cause a reduction in growth as measured by height when administered to infants, as suggested by observations from the controlled study. It is unknown whether any effect of SPINRAZA on growth would be reversible with cessation of treatment.

The most common adverse reactions (≥20% of SPINRAZA-treated patients and ≥5% more frequently than in control patients) that occurred in the infantile-onset controlled study were lower respiratory infection and constipation. Serious adverse reactions of atelectasis were more frequent in SPINRAZA-treated patients (18%) than in control patients (10%). Because patients in this controlled study were infants, adverse reactions that are verbally reported could not be assessed. The most common adverse reactions that occurred in the later-onset controlled study were pyrexia, headache, vomiting, and back pain. Post-lumbar puncture syndrome has also been observed after the administration of SPINRAZA.

Please see full Prescribing Information for additional Important Safety Information.

As a courtesy, our full Prescribing Information is also available en Español. For prescribing decisions, please refer to official approved labeling.