INDICATION
SPINRAZA® (nusinersen) 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.
In the sham-controlled studies for patients with infantile-onset and later-onset SMA, 24 of 146 SPINRAZA-treated patients (16%) with high, normal, or unknown platelet count at baseline developed a
platelet level below the lower limit of normal, compared to 10 of 72 sham-controlled patients (14%). Two
SPINRAZA-treated patients developed platelet counts <50,000 cells per microliter, with the lowest level of 10,000 cells per microliter recorded on study day 28.
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
the sham-controlled studies for patients with infantile-onset and later-onset SMA, 71 of 123
SPINRAZA-treated patients (58%) had elevated urine protein, compared to 22 of 65 sham-controlled
patients (34%).
Laboratory testing and monitoring to assess safety should be conducted. Perform a
platelet count, coagulation laboratory testing, and quantitative spot urine protein testing at baseline
and prior to each dose of SPINRAZA and as clinically needed.
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 (≥20% of SPINRAZA-treated patients and ≥5% more
frequently
than in control patients) that occurred in the infantile-onset controlled study were lower respiratory
infection and constipation. Serious adverse reactions of atelectasis were more frequent in
SPINRAZA-treated patients (18%) than in control patients (10%). Because patients in this controlled
study were infants, adverse reactions that are verbally reported could not be assessed. The most common
adverse reactions that occurred in the later-onset controlled study were pyrexia, headache, vomiting,
and back pain. Post-lumbar puncture syndrome has also been observed after the administration of
SPINRAZA.
Please see full Prescribing Information.
