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Frequently occurring adverse reactions

Adverse reactions (ARs) that occurred in ≥5% of patients treated with SPINRAZA and ≥5% more frequently or ≥2 times as frequently than in control patients in ENDEAR1


ARs SPINRAZA*
(n=80)
Untreated
(n=41)
Lower respiratory infection 43% 29%
Upper respiratory infection 39% 34%
Constipation 30% 22%
Teething 14% 7%
Upper respiratory tract congestion 6% 2%
Aspiration 5% 2%
Ear infection 5% 2%
Scoliosis 5% 2%

*Four loading doses followed by 12 mg (5 mL) once every 4 months.
Includes pneumonia, bronchiolitis, pneumonia viral, respiratory syncytial virus bronchiolitis, lower respiratory tract infection, pneumonia bacterial, bronchitis, bronchitis viral, pneumonia moraxella, pneumonia parainfluenzae viral, lower respiratory tract infection viral, lung infection, pneumonia influenza, pneumonia pseudomonal, and pneumonia respiratory syncytial viral.
Includes upper respiratory tract infection, nasopharyngitis, rhinitis, pharyngitis, or tracheitis.


Additional Safety Considerations1

  • Serious ARs of atelectasis were more frequent (14%) in patients treated with SPINRAZA than in control patients (5%)1
  • The most common AEs in the open-label studies in later-onset patients were headache (50%), back pain (41%), and post lumbar puncture syndrome (41%). Most of these events occurred within 5 days of lumbar puncture. Other AEs in these patients were consistent with ARs observed in the controlled study1

  • Cases of rash were reported in patients treated with SPINRAZA. One patient, 8 months after starting SPINRAZA treatment, developed painless red macular lesions on the forearm, leg, and foot over an 8-week period. The lesions ulcerated and scabbed over within 4 weeks, and resolved over several months. A second patient developed red macular skin lesions on the cheek and hand 10 months after the start of SPINRAZA treatment, which resolved over 3 months
    • Both cases continued to receive SPINRAZA and had spontaneous resolution of the rash
  • SPINRAZA may cause a reduction in growth as measured by height when administered to infants. It is unknown whether any effect of SPINRAZA on growth is reversible with cessation of treatment
  • In an open-label clinical study in infants with symptomatic SMA, severe hyponatremia was reported in a patient treated with SPINRAZA requiring salt supplementation for 14 months

Photo of Sofia aged 2.5 years old with Type 1 SMA looking out window
Sofia
age 2.5

Infantile-onset (Type 1) SMA treated with SPINRAZA

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

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.