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Frequently occurring adverse reactions (ARs) in ENDEAR

ARs that occurred in ≥5% of patients treated with SPINRAZA and ≥5% more frequently or ≥2 times as frequently than in control patients1

ARs SPINRAZA*
(n=80)
Untreated
(n=41)
Lower respiratory infection 55% 37%
Constipation 35% 22%
Teething 18% 7%
Urinary tract infection 9% 0%
Upper respiratory tract congestion 8% 2%
Ear infection 6% 2%
Flatulence 5% 2%
Decreased weight 5% 2%

*Loading doses followed by 12 mg (5 mL) once every 4 months.1
Includes adenovirus infection, bronchiolitis, bronchitis, bronchitis viral, corona virus infection, influenza, lower respiratory tract infection, lower respiratory tract infection viral, lung infection, parainfluenzae virus infection, pneumonia, pneumonia bacterial, pneumonia influenzal, pneumonia moraxella, pneumonia parainfluenzae viral, pneumonia pneumococcal, pneumonia pseudomonal, pneumonia respiratory syncytial viral, pneumonia viral, and respiratory syncytial virus bronchiolitis.1


Frequently occurring ARs in CHERISH

ARs that occurred in ≥5% of patients treated with SPINRAZA and ≥5% more frequently or ≥2 times as frequently than in control patients1

ARs SPINRAZA
(n=84)
Untreated
(n=42)
Pyrexia 43% 36%
Headache 29% 7%
Vomiting 29% 12%
Back pain 25% 0%
Epistaxis 7% 0%
Fall 5% 0%
Respiratory tract congestion 5% 2%
Seasonal allergy 5% 2%

Loading doses followed by 12 mg (5 mL) once every 6 months.1

Additional safety considerations1

Additional ARs

  • Serious ARs of atelectasis were more frequent (18%) in patients treated with SPINRAZA than in control patients (10%)
  • 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 would be 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

Postmarketing events

  • The following ARs have been identified during post-approval use of SPINRAZA. Because these reactions are reported voluntarily from a population of uncertain size, it is not always possible to reliably estimate their frequency or establish a causal relationship to drug exposure
  • Serious infections (including meningitis) and hydrocephalus have occurred in patients treated with SPINRAZA via lumbar puncture

Photo of Ruby aged 4 years old with Type 2 SMA
Ruby
age 4

Later-onset (Type 2) 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.

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 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.