The safety profile of SPINRAZA® (nusinersen)

Warnings and precautions1

Coagulation abnormalities and thrombocytopenia, including acute severe thrombocytopenia, have been observed after administration of some antisense oligonucleotides (ASOs). In the sham-controlled studies for patients with infantile-onset and later-onset spinal muscular atrophy (SMA), 24 of 146 (16%) SPINRAZA-treated patients with high, normal, or unknown platelet count at baseline developed a platelet level below the lower limit of normal, compared to 10 of 72 (14%) sham-controlled patients. Because of the risk of thrombocytopenia and coagulation abnormalities from SPINRAZA, patients may be at increased risk of bleeding complications.

Renal toxicity, including potentially fatal glomerulonephritis, has been observed after administration of some ASOs. 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 (58%) of SPINRAZA-treated patients had elevated urine protein, compared to 22 of 65 (34%) sham-controlled patients. 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 greater than 0.2 g/L, consider repeat testing and further evaluation. Please see lab testing and monitoring information below.

Laboratory testing and monitoring information1

Due to the risk of coagulation abnormalities, thrombocytopenia, and renal toxicity, the following laboratory tests are recommended at baseline and prior to each dose of SPINRAZA and as clinically needed:

Icon of three test tubes
Icon of three test tubes

Platelet count

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Icon of clock

Prothrombin time; activated partial thromboplastin time

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Icon of checklist

Quantitative spot
urine protein testing

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.

SPINRAZA is delivered as a 12-mg (5-mL) dose at each injection and is administered intrathecally.1

Bill
age 48

Later-onset (Type 3) SMA
treated with SPINRAZA

 

 

Bill
age 48

Later-onset (Type 3) SMA
treated with SPINRAZA

 

 

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