WHY SPINRAZA? / ABOUT SPINRAZA

SPINRAZA (12 mg):

The first-ever FDA-approved treatment for SMA1,2
SPINRAZA is a treatment option approved for all spinal muscular atrophy (SMA)1
  • Approved for all ages–adults, teens, children, and infants
  • Approved for all SMA, regardless of time of onset
  • Approved for all SMA types
See Efficacy
Specifically targets and increases survival motor neuron (SMN) protein production1
  • Targets a specific sequence on SMN2 mRNA transcripts to increase exon 7 inclusion and production of full-length SMN protein1
  • The first and only antisense oligonucleotide (ASO) SMA treatment1-4
  • Delivered directly to the cerebrospinal fluid (CSF) for distribution throughout the central nervous system (CNS) where cell bodies of lower motor neurons live1,5
  • Long half-life (~135-177 days) in the CSF maintains a high concentration in between prescribed doses1
See the MOA
Delivered only 3x a year after a loading phase1
  • Loading phase: doses 1-3 administered at 14-day intervals; dose 4 administered 30 days after dose 3
  • Maintenance doses: administered once every 4 months
  • Administered by an HCP experienced in performing lumbar punctures
See Dosing
Validated in pivotal trials. Supported in real-world studies
  • The longest clinical trial program of SMA treatment to date6
  • Real-world studies with one up to 38 months of treatment and in adults up to 71 years old7
See Data
Well-established safety profile1

Warnings and precautions:

  • Thrombocytopenia and coagulation abnormalities: increased risk for bleeding complications; testing required at baseline and before each dose and as clinically needed
  • Renal toxicity: quantitative spot urine protein testing required at baseline and prior to each dose
See Safety
A diverse community of SMA patients worldwide8
  • More than 14,000 patients treated, including 5,300+ adults8*
  • From infants to adults, 1 day old to 85 years old9†‡
  • Hundreds of patients have restarted SPINRAZA10
See More

*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.1

ASO, antisense oligonucleotide; CNS, central nervous system; CSF, cerebrospinal fluid; FDA, Food and Drug Administration; HCP, healthcare professional; MOA, mechanism of action; mRNA, messenger ribonucleic acid; SMA, spinal muscular atrophy; SMN, survival motor neuron; SMN2, survival motor neuron 2 gene.

Review the warnings and precautions, including thrombocytopenia, coagulation abnormalities, and renal toxicity1

See spinraza safety
Start your patient on spinraza

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. SPINRAZA. Prescribing Information. Biogen; 2024. 2. FDA approves first drug for spinal muscular atrophy [press release]. Silver Spring, MD: U.S. Food and Drug Administration; December 23, 2016. Accessed September 5, 2024. https://www.fda.gov/news-events/press-announcements/fda-approves-first-drug-spinal-muscular-atrophy 3. Evrysdi. Prescribing Information. Genentech; 2024. 4. Zolgensma. Prescribing Information. Novartis; 2024. 5. Zayia LC, Tadi P. Neuroanatomy, motor neuron. [Updated 2023 Jul 24]. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2024 Jan. Available from: https://ncbi.nlm.nih.gov/books/NBK554616/ 6. Biogen Data on File as of 9/21. 7. 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 8. Biogen Data on File as of 7/24. 9. Biogen Data on File as of 5/24. 10. Biogen Data on File as of 1/24.

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