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With SPINRAZA, some patients achieved milestones they would not be expected to achieve, such as:

“All these new mobilities that she has were things that we were told she would never be able to do.”

–Sofia’s mother

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

In open-label studies treating patients for up to 3 years, some patients sustained improvements in milestones at ages when they would otherwise be lost.1,2

  • At least 1 of 11 patients with Type 2 SMA gained the ability to walk independently2
  • At least 2 of 4 patients with Type 3 SMA regained the ability to walk independently2

SPINRAZA helps maintain motor function in later-onset SMA

The efficacy of SPINRAZA was demonstrated in ENDEAR and supported by an open-label study in patients with later-onset (Type 2 and Type 3) spinal muscular atrophy who maintained and increased motor function.1

Change in Hammersmith Functional Motor Scale Expanded (HFMSE) score from baseline after 1050 days2

The primary objective of this study was to examine the safety and tolerability of SPINRAZA in patients with Type 2 or Type 3 SMA. Additional exploratory endpoints included HFMSE, ULM (nonambulatory patients), and 6MWT (ambulatory patients).

Limitations of this study include that it was a longitudinal roll-over, re-dose, open-label study of 28 patients who received their fi rst dose in CS2 and then transferred to the second phase, CS12. Altogether, the dosage (3 mg-12 mg in CS2) and administration schedule in these studies differed from the approved SPINRAZA regimen.2

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

Patients may walk farther with SPINRAZA

In an open-label study, some ambulatory patients with later-onset spinal muscular atrophy were able to increase their walking distance in the 6-Minute Walk Test (6MWT) with SPINRAZA.2

Change in 6MWT distance from baseline after 1050 days2

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In a natural history cohort, there was no significant change in 6MWT at 12 months.4

Photo of Emma aged 7 years old with Type 3 SMA playing with a ribbon
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.