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Primary endpoint in ENDEAR:

Patients who showed a substantial motor milestone response on their Hammersmith Infant Neurological Examination (HINE) Section 2 score in the interim efficacy set (IES)1

ENDEAR is a phase 3, multicenter, randomized, double-blind, sham procedure–controlled study in 121 symptomatic infants ≤7 months of age at the time of first dose who were diagnosed with SMA (symptom onset before 6 months of age). Patients were randomized 2:1 to either SPINRAZA or untreated groups.1

SPINRAZA may unlock new abilities

Some patients with infantile-onset (Type 1) spinal muscular atrophy (SMA) treated with SPINRAZA achieved new motor milestones.1

Patients achieving improvements in HINE Section 2 motor milestone categories in the IES2*

A planned interim efficacy analysis was conducted based on 82 patients who completed at least 183 days of treatment, withdrew from the study, or died. For subjects who were alive and ongoing in the study, the change in total motor milestone score was calculated at the later of days 183, 302, or 394.

*Voluntary grasp, a category in which none of the milestones require movement against gravity, was excluded from the analysis as some infants with SMA can acquire all milestones in this category.2

Patients achieving ≥2-point improvement or reaching the maximal score.

Key Icon

Milestones such as head control, rolling, and sitting, are rarely, if ever, achieved in untreated patients in the natural history of Type 1 SMA.3

Potential milestone gains with SPINRAZA

Patients with infantile-onset (Type 1) spinal muscular atrophy (SMA) treated with SPINRAZA saw improvements in motor milestones.1

Net change from baseline in HINE Section 2 total motor milestone score in the IES1

A planned interim efficacy analysis was conducted based on 82 patients who completed at least 183 days of treatment, withdrew from the study, or died. For subjects who were alive and ongoing in the study, the change in total motor milestone score was calculated at the later of days 183, 302, or 394.

Key Icon

All patients treated with SPINRAZA who were alive at the time of the interim efficacy analysis either maintained or improved their HINE Section 2 scores.1

Photo of Sofia aged 2.5 years old with Type 1 SMA playing in the bath
Sofia
age 2.5

Infantile-onset (Type 1)
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.