SPINRAZA (nusinersen sodium)

AbsorptionIntrathecal injection of SPINRAZA into the cerebrospinal fluid (CSF) allows nusinersen to be distributed from the CSF to the target central nervous system (CNS) tissues. Following intrathecal administration, trough plasma concentrations of nusinersen were relatively low, compared to the trough CSF concentration. Median plasma T max values ranged from 1.7 to 6.0 hours. Mean plasma C max and AUC values increased approximately dose-proportionally up to a dose of 12 mg.

DistributionAutop...

The efficacy of SPINRAZA was demonstrated in two double-blind, sham-procedure controlled clinical trials in symptomatic infantile-onset and later-onset SMA patients (Study 1 and Study 2) and was supported by open-label clinical trials conducted in presymptomatic and symptomatic SMA patients. The overall findings from these trials support the effectiveness of SPINRAZA across the range of SMA patients, and appear to support the early initiation of treatment with SPINRAZA. 14.1 Infantile-Onset SMA

Study 1(NCT02193074) was a multicenter, randomized, double-blind, sham-procedure controlled study i...

The following serious adverse reactions are described in detail in other sections of the labeling: Thrombocytopenia and Coagulation Abnormalities [see Warnings and Precautions ( 5.1 )] Renal Toxicity [see Warnings and Precautions ( 5.2 )] The most common adverse reactions that occurred in at least 20% of SPINRAZA-treated patients and occurred at least 5% more frequently than in control patients were: lower respiratory infection and constipation in patients with infantile-onset SMA ( 6.1 ) pyrexi...

None. None.

Thrombocytopenia and Coagulation Abnormalities: Increased risk for bleeding complications; testing required at baseline and before each dose and as clinically needed ( 5.1 , 2.3 ) Renal Toxicity: Quantitative spot urine protein testing required at baseline and prior to each dose ( 5.2 , 2.3 ) 5.1 Thrombocytopenia and Coagulation Abnormalities Coagulation abnormalities and thrombocytopenia, including acute severe thrombocytopenia, have been observed after administration of some antisense oligonuc...