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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. Perform a platelet count and coagulation laboratory testing at baseline and prior to each administration of SPINRAZA and as clinically needed.

In a clinical study, 11% of SPINRAZA-treated patients with normal or above normal platelet levels at baseline developed a platelet level below the lower limit of normal compared to zero sham-procedure control patients. No patient had a platelet count <50,000 cells per mcL and no patient developed a sustained low platelet count despite continued drug exposure.

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 a clinical study (mean treatment exposure 7 months), 33% of SPINRAZA-treated patients had elevated urine protein, compared to 20% of sham-control patients. In a group of later-onset SMA patients (mean treatment exposure 34 months), 69% had elevated urine protein.

No elevations in serum creatinine or cystatin C were observed in studies with SPINRAZA. Conduct quantitative spot urine protein testing (preferably using a first morning urine specimen) at baseline and prior to each dose of SPINRAZA. For urinary protein concentration >0.2 g/L, consider repeat testing and further evaluation.

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 that occurred in the controlled study in at least 20% of SPINRAZA-treated patients and occurred at least 5% more frequently than in control patients were upper respiratory infection (39% vs 34%), lower respiratory infection (43% vs 29%), and constipation (30% vs 22%). Serious adverse reactions of atelectasis were more frequent in SPINRAZA-treated patients (14%) than in control patients (5%). Because patients in the controlled study were infants, adverse reactions that are verbally reported could not be assessed in this study. In the open-label studies, the most common adverse events in later onset patients were headache (50%), back pain (41%) and post lumbar puncture syndrome (41%).

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.