WHY SPINRAZA/HOW SPINRAZA WORKS

SMA is a disease of the central nervous system (CNS). SPINRAZA is delivered directly to the CNS1-3

Individuals with SMA have a mutated survival motor neuron 1 (SMN1) gene and rely on the SMN2 gene to produce functional SMN protein. But the SMN2 gene can only produce about 10% of the full-length, functional protein that motor neurons need.1,3

As an antisense oligonucleotide (ASO), SPINRAZA targets an underlying cause of motor neuron loss by binding to a specific sequence in the SMN2 gene to increase the production of functional SMN protein in the CNS.3,4

Delivery of SPINRAZA to the CNS helps infuse the motor neurons with nusinersen and maintain an adequate concentration and long half-life in the spinal cord, where it's needed most3,5,6

mRNA=messenger ribonucleic acid.

ASOs like SPINRAZA bind to specific nucleotide sequences. SPINRAZA binds to a sequence in the intron downstream of exon 7 of the SMN2 transcript3

Watch how SPINRAZA works to enhance the production of full-length SMN protein3

Download the patient materials to talk about SPINRAZA today.

SPINRAZA
Pediatric Brochure

Information
for Adults

Did you know SPINRAZA is indicated
for both
pediatric and adult
patients with SMA?3

Explore later-onset efficacy
Start your patient on SPINRAZA

INDICATION

SPINRAZA 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. Darras BT, Royden Jones H Jr, Ryan MM, De Vivo DC, eds. Neuromuscular Disorders of Infancy, Childhood, and Adolescence: A Clinician’s Approach. 2nd ed. London, UK: Elsevier; 2015. 2. National Institutes of Health, National Library of Medicine. MedlinePlus: SMN1 gene. Updated August 18, 2020. Accessed March 7, 2023. ghr.nlm.nih.gov/gene/SMN1. 3. SPINRAZA. Prescribing Information. Biogen; 2023. 4. Lunn MR, Wang CH. Spinal muscular atrophy. Lancet. 2008;371(9630):2120-2133. 5. European Medicines Agency. Assessment report: Spinraza International non-proprietary name: nusinersen 2017. 6. Food and Drug Administration, Center for Drug Evaluation and Research. Pharmacology/toxicology review and evaluation. White Oak, MD; 2016.

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