In a SPINRAZA® (nusinersen) 12-mg pivotal trial, people with later-onset spinal muscular atrophy (SMA) showed meaningful improvements in overall motor function, as measured by mean Hammersmith Functional Motor Scale—Expanded (HFMSE), compared to placebo at 15 months. Additionally, in a 12-mg pivotal trial in people with early-onset SMA, a majority of infants were motor milestone responders at 13 months.

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

These patients are paid spokespersons for Biogen.

*Pivotal trials did not include adult patients with SMA.

Learn more about important clinical trials and real-world trial data

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How Does SPINRAZA Work?

Explore the MOA

An antisense oligonucleotide delivered directly to the central nervous system (CNS) to target an underlying cause of motor neuron loss in SMA3,8
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CNS, central nervous system; MOA, mechanism of action; SMA, spinal muscular atrophy.

Extensive real-world experience from infants to adults across disease severities9,10†‡§

Including pediatric to adult, hundreds of patients have restarted SPINRAZA11

have been treated with SPINRAZA worldwide9†

have been treated with SPINRAZA worldwide9†

there’s someone from almost every age group who has taken SPINRAZA10‡§  

†Based on commercial patients and early access patients through June 2024.

‡Based on patients treated with SPINRAZA in the US through May 2024.

§SPINRAZA (12 mg) clinical studies included patients from 3 days to 16 years of age at first dose and did not include sufficient numbers of subjects aged 65 and older to determine whether they respond differently from younger patients. Pivotal studies did not include adult patients.3

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.

As a courtesy, our full Prescribing Information is also available en Español. For prescribing decisions, please refer to official approved labeling.

References

1. Biogen Data on File as of 1/24. 2. De Vivo DC, Bertini E, Swoboda KJ, et al; NURTURE Study Group. Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: interim efficacy and safety results from the Phase 2 NURTURE study. Neuromuscul Disord. 2019;29(11):842-856. 3. SPINRAZA. Prescribing Information. Biogen; 2024. 4. Finkel RS, Mercuri E, Darras BT, et al; ENDEAR Study Group. Nusinersen versus sham control in infantile-onset spinal muscular atrophy. N Eng J Med. 2017;377(18):1723-1732. 5. Mercuri E, Darras BT, Chiriboga CA, et al; CHERISH Study Group. Nusinersen versus sham control in later-onset spinal muscular atrophy. N Eng J Med. 2018;378(7):625-635. 6. Hagenacker T, Wurster CD, Günther R, et al. Appendix to: Nusinersen in adults with 5q spinal muscular atrophy: a non-interventional, multicentre, observational cohort study. Lancet Neurol. 2020;19(4):317-325. 7. Crawford TO, Kirschner J, Ryan MM, et al; NURTURE Study Group. Nusinersen effect in infants in the presymptomatic stage of SMA: 4.9-year interim of the NURTURE study. Presented at: Muscular Dystrophy Association (MDA) 4th Clinical and Scientific Conference. March 13–16, 2022; Nashville, TN. 8. Lunn MR, Wang CH. Spinal muscular atrophy. Lancet. 2008;371:2120-2133. 9. Biogen Data on File as of 7/24. 10. Biogen Data on File as of 5/24. 11. Biogen Data on File as of 1/24.

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