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    The first and only treatment for pediatric and adult patients with spinal muscular atrophy (SMA)1

    The first and only treatment for pediatric and adult patients with spinal muscular atrophy (SMA)1

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

    DISCOVER THE BENEFITS OF SPINRAZA

    that may be possible for individuals with SMA

    REVIEW THE SPINRAZA SAFETY PROFILE

    for warnings and precautions, monitoring information, and adverse reactions

    DOWNLOAD THE START FORM

    to help your patients get started and get support with SPINRAZA

    FIND OUT HOW SPINRAZA IS REIMBURSED

    for individuals with SMA
    Use the SPINRAZA Treatment Locator to help your patients find a treatment facility

    USE THE SPINRAZA TREATMENT LOCATOR

    to help patients and caregivers find a treatment facility nearby
    Effective January 1, 2018, J2326 is the permanent J-code for SPINRAZA

    PERMANENT J-CODE ASSIGNED FOR SPINRAZA

    J2326 is the permanent J-code for SPINRAZA, effective January 1, 2018

    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. Perform a platelet count and coagulation laboratory testing at baseline and prior to each administration of SPINRAZA and as clinically needed.

    In a clinical study, 11% of SPINRAZA-treated patients with normal or above normal platelet levels at baseline developed a platelet level below the lower limit of normal compared to zero sham-procedure control patients. No patient had a platelet count <50,000 cells per mcL and no patient developed a sustained low platelet count despite continued drug exposure.

    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 a clinical study (mean treatment exposure 7 months), 33% of SPINRAZA-treated patients had elevated urine protein, compared to 20% of sham-control patients. In a group of later-onset SMA patients (mean treatment exposure 34 months), 69% had elevated urine protein.

    No elevations in serum creatinine or cystatin C were observed in studies with SPINRAZA. Conduct quantitative spot urine protein testing (preferably using a first morning urine specimen) at baseline and prior to each dose of SPINRAZA. For urinary protein concentration >0.2 g/L, consider repeat testing and further evaluation.

    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 that occurred in the controlled study in at least 20% of SPINRAZA-treated patients and occurred at least 5% more frequently than in control patients were upper respiratory infection (39% vs 34%), lower respiratory infection (43% vs 29%), and constipation (30% vs 22%). Serious adverse reactions of atelectasis were more frequent in SPINRAZA-treated patients (14%) than in control patients (5%). Because patients in the controlled study were infants, adverse reactions that are verbally reported could not be assessed in this study. In the open-label studies, the most common adverse events in later onset patients were headache (50%), back pain (41%) and post lumbar puncture syndrome (41%).

    Please see full Prescribing Information for additional Important Safety Information.

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

    REFERENCES

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    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. Perform a platelet count and coagulation laboratory testing at baseline and prior to each administration of SPINRAZA and as clinically needed.

    In a clinical study, 11% of SPINRAZA-treated patients with normal or above normal platelet levels at baseline developed a platelet level below the lower limit of normal compared to zero sham-procedure control patients. No patient had a platelet count <50,000 cells per mcL and no patient developed a sustained low platelet count despite continued drug exposure.

    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 a clinical study (mean treatment exposure 7 months), 33% of SPINRAZA-treated patients had elevated urine protein, compared to 20% of sham-control patients. In a group of later-onset SMA patients (mean treatment exposure 34 months), 69% had elevated urine protein.

    No elevations in serum creatinine or cystatin C were observed in studies with SPINRAZA. Conduct quantitative spot urine protein testing (preferably using a first morning urine specimen) at baseline and prior to each dose of SPINRAZA. For urinary protein concentration >0.2 g/L, consider repeat testing and further evaluation.

    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 that occurred in the controlled study in at least 20% of SPINRAZA-treated patients and occurred at least 5% more frequently than in control patients were upper respiratory infection (39% vs 34%), lower respiratory infection (43% vs 29%), and constipation (30% vs 22%). Serious adverse reactions of atelectasis were more frequent in SPINRAZA-treated patients (14%) than in control patients (5%). Because patients in the controlled study were infants, adverse reactions that are verbally reported could not be assessed in this study. In the open-label studies, the most common adverse events in later onset patients were headache (50%), back pain (41%) and post lumbar puncture syndrome (41%).

    Please see full Prescribing Information for additional Important Safety Information.

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