WHY SPINRAZA? / ABOUT SPINRAZA

The legacy of the first FDA-approved treatment for SMA, now including SPINRAZA® (nusinersen)
High Dose Regimen1,2
SPINRAZA is a treatment option approved for spinal muscular atrophy1
  • Approved for all ages–adults, teens, children, and infants
  • Approved for all SMA types, regardless of time of onset

Efficacy of High Dose SPINRAZA3

  • Evaluated in the DEVOTE study
  • Outcomes measured include motor function scores, biomarkers, and survival
See MOA
SPINRAZA High Dose Regimen was studied in the 3-part DEVOTE trial3
  • DEVOTE evaluated the safety and efficacy of High Dose SPINRAZA in infantile-onset and later-onset patients
  • DEVOTE Part B examined High Dose SPINRAZA in treatment-naive patients with symptomatic SMA
  • DEVOTE Part C examined High Dose SPINRAZA in patients who had been previously treated with Low Dose SPINRAZA for a median of 3.9 years
Explore DEVOTE Results
High Dose SPINRAZA is approved1

SPINRAZA High Dose Regimen

  • Patients who are prescribed High Dose SPINRAZA receive two 50 mg loading doses 14 days apart, followed by a 28 mg maintenance dose every 4 months thereafter
  • SPINRAZA is administered intrathecally by, or under the direction of, healthcare professionals experienced in performing lumbar punctures
See Dosing
Validated in Low Dose SPINRAZA pivotal trials. Supported in real-world studies1,4-6

SPINRAZA Low Dose Regimen

  • The longest clinical trial program of any SMA treatment to date7
  • Real-world studies, with one up to 38 months of treatment and in adults up to 71 years old6
See Data
The safety profile of SPINRAZA is supported by clinical trial data collected over 10+ years1,5-7

Warnings and Precautions:

  • Thrombocytopenia and coagulation abnormalities: increased risk for bleeding complications; testing required at baseline and before each dose and as clinically needed1
  • Renal toxicity: quantitative spot urine protein testing required at baseline and prior to each dose1
See Safety
A diverse community of SMA patients on SPINRAZA8
  • ~10 years of treating patients1
  • Patients treated with SPINRAZA in the US have a collective ~16,700 years of treatment experience9*
  • From 0 days to 86 years old, patients across a broad range of ages have taken SPINRAZA8†‡
See More

*Based on consented patients from a real-world sample in the US who are or have ever been treated with SPINRAZA through April 2026.9

†Based on patients treated with SPINRAZA in the US through April 2026.8

‡Pivotal studies did not include adult patients. Clinical studies did not include sufficient numbers of subjects aged 65 and over to determine whether they respond differently than younger subjects.1

FDA, Food and Drug Administration; SMA, spinal muscular atrophy.

The overall findings from clinical trials support the early initiation of treatment with SPINRAZA.1

Learn about an SMA Treatment
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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 (Study 1) and later-onset (Study 2) SMA who received Low Dose Regimen (12 mg loading doses/12 mg maintenance doses), 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. In patients who received High Dose Regimen (50 mg loading doses/28 mg maintenance doses), decreases in platelet counts were also observed.

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 Study 1 and Study 2, 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 in the Low Dose Regimen (≥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.

The most common adverse reactions in the High Dose Regimen (≥10% of SPINRAZA-treated patients and ≥5% more frequently than control patients from Study 1) that occurred in patients with infantile-onset SMA were pneumonia, COVID-19, pneumonia aspiration, and malnutrition. COVID-19 was not discovered at the time of Study 1.

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. SPINRAZA. Prescribing Information. Biogen; 2026. 2. U.S. FDA approves Biogen’s SPINRAZA™ (nusinersen), the first treatment for spinal muscular atrophy [press release]. Biogen. December 23, 2016. Accessed March 19, 2026. https://investors.biogen.com/news-releases/news-release-details/us-fda-approves-biogens-spinrazatm-nusinersen-first-treatment 3. Finkel RS, Crawford TO, Mercuri E, et al. High-dose nusinersen for spinal muscular atrophy: a phase 3 randomized trial. Nat Med. 2026;32(3):1095-1104. doi:10.1038/s41591-025-04193-6 4. Hagenacker T, Paradis AD, Lawson-Michod KA, Youn B. Systematic review and meta-analysis of long-term nusinersen effectiveness in adolescents and adults with spinal muscular atrophy. Adv Ther. 2025;42(9):4143-4160. doi:10.1007/s12325-025-03260-1 5. Łusakowska A, Wójcik A, Frączek A, et al. Long-term nusinersen treatment across a wide spectrum of spinal muscular atrophy severity: a real-world experience. Orphanet J Rare Dis. 2023;18(1):230. doi:10.1186/s13023-023-02769-4 6. Günther R, Wurster CD, Brakemeier S, et al. Long-term efficacy and safety of nusinersen in adults with 5q spinal muscular atrophy: a prospective European multinational observational study. Lancet Reg Health Eur. 2024;39:100862. doi:10.1016/j.lanepe.2024.100862 7. Biogen, Data on File as of 06/26. 8. Biogen, Data on File as of 04/26. 9. Biogen, Data on File as of 04/26.

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