WHY SPINRAZA? / HOW SPINRAZA WORKS

Works within motor neurons to increase
full-length SMN protein1

SPINRAZA is the first antisense oligonucleotide (ASO) for the treatment
of spinal muscular atrophy (SMA).1,2

Designed to target a specific sequence on survival motor neuron 2 (SMN2)
pre-mRNA, SPINRAZA modifies splicing to increase exon 7 inclusion and
production of full-length SMN protein1,3

Mechanism of Action Delivered to the CNS Video Resource Download

By specifically targeting SMN2 to increase SMN protein, SPINRAZA works by targeting an underlying cause of CNS motor neuron loss in SMA1,4

  • SMA is a disease of the central nervous system (CNS) leading to progressive weakness4,5
  • Individuals with SMA have a mutated survival motor neuron 1 gene, or SMN1, and rely only on the SMN2 gene to produce functional SMN protein4,5
  • However, the SMN2 gene by itself can only produce about 10% of the full-length, functional protein that is normally produced by SMN1 4,5
  • SPINRAZA is an ASO designed to target a specific sequence on SMN2 pre-mRNA
  • SPINRAZA binds to a sequence in the intron downstream of exon 7 in the SMN2 transcript to modify splicing
  • Increasing exon 7 inclusion in SMN2 mRNA transcripts results in increased production of full-length SMN protein

ASO, antisense oligonucleotide; CNS, central nervous system; mRNA, messenger ribonucleic acid; SMA, spinal muscular atrophy; SMN, survival motor neuron; SMN1, survival motor neuron 1 gene; SMN2, survival motor neuron 2 gene.

As an ASO, SPINRAZA is delivered directly to the CNS1,3

Intrathecal delivery concentrates nusinersen in the cerebrospinal fluid (CSF)
and bypasses the blood-brain barrier
Targets CNS tissues1
  • Distributed to where cell bodies of lower motor neurons live1,6
  • Infuses motor neurons with nusinersen1
Persists in target tissues1
  • Maintains high concentration and long half-life in the CSF between prescribed doses
  • Half-life of up to 5.9 months (estimated to be 135-177 days)
Consistent drug exposure across ages and weights7
  • Biogen pharmacokinetic Data on File from 2-15 years of age

ASO, antisense oligonucleotide; CNS, central nervous system; CSF, cerebrospinal fluid.

Watch how SPINRAZA works to enhance the production of
full-length SMN protein1

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

SEE SPINRAZA SAFETY

Did you know SPINRAZA is the first and only ASO treatment for SMA?1,2,9,10

Download the resource below to learn more about SPINRAZA as an ASO, intrathecal administration, and common questions from patients to help prepare them for their injection.
SPINRAZA Intrathecal BrochureSPINRAZA ASO and Intrathecal Administration Brochure

ASO, antisense oligonucleotide; SMA, spinal muscular atrophy; SMN, survival motor neuron.

SPINRAZA is approved for all ages—adults,
teens, children, and infants1

EXPLORE LATER-ONSET EFFICACY
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. Chiriboga CA, Swoboda KJ, Darras BT, et al. Results from a phase 1 study of nusinersen (ISIS-SMNRx) in children with spinal muscular atrophy. Neurology. 2016;86:1-8. 4. Lunn MR, Wang CH. Spinal muscular atrophy. Lancet. 2008;371:2120-2133. 5. Darras BT, Royden Jones Jr H, Ryan MM, De Vivo DC, eds. Neuromuscular Disorders of Infancy, Childhood, and Adolescence: A Clinician’s Approach. 2nd ed. London, UK: Elsevier; 2015. 6. 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/ 7. Biogen Data on File as of 9/21. 8. Haché M, Swoboda KJ, Sethna N, et al. Intrathecal injections in children with spinal muscular atrophy: nusinersen clinical trial experience. J Child Neurol. 2016;31(7):899-906. 9. Evrysdi. Prescribing Information. Genentech; 2024. 10. Zolgensma. Prescribing Information. Novartis; 2024.

Stay Connected
Sign up with us to receive news and support information and hear about upcoming events.

ABOUT SMA
What Is SMA? Multidisciplinary Care
Why SPINRAZA?
About SPINRAZA How SPINRAZA Works Presymptomatic Efficacy Early-Onset Efficacy Later-Onset Efficacy Real-World Evidence Safety
Starting SPINRAZA
How to Start Dosing & Administration
Patients on Therapy
Patients on Therapy
RESOURCE LIBRARY
Access & Reimbursement SMA360°™ Patient Support Events Video Hub

©2025 Biogen. All rights reserved.
This site is intended for US Healthcare Professionals only.
SPZ-US-3383 v11 02/25