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WHY SPINRAZA/About SPINRAZA

SPINRAZA has been studied for 7+ years in the longest clinical trial program in SMA to date1

Studies have evaluated the effectiveness of SPINRAZA for improving survival, overall motor function, and walking ability1,2

TRIALS FOR LATER-ONSET SMA

Pivotal trial: CHERISH2,3

Study: A phase 3, multicenter, randomized (2:1), double-blind, sham procedure– controlled trial

Treatment duration: 15 months

Participants: 126 patients with later-onset SMA aged 2 to 9 years at screening

Primary endpoint: Change in motor function as measured by the Hammersmith Functional Motor Scale—Expanded (HFMSE)

Safety: The most common side effects were fever (43%), headache (29%), vomiting (29%), and back pain (25%)

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Supportive trial: CS2/CS124

Study: A phase 1b/2a, open-label, multiple-dose, dose-escalation integrated analysis of CS2 and CS12 trials

Treatment duration: ~3 years (38 months)

Participants: 28 ambulatory and nonambulatory patients with SMA aged 2 to 16 years at time of first dose

Primary endpoint: Safety of SPINRAZA

Safety: Adverse reactions were similar to those reported in the pivotal trials

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TRIALS FOR EARLY-ONSET AND PRESYMPTOMATIC SMA

Pivotal trial: ENDEAR2,5

Study: A phase 3, multicenter, double-blind, randomized (2:1), sham-controlled trial

Treatment duration: 13 months

Participants: 121 patients with early-onset SMA aged ≤7 months at time of first dose

Primary endpoints:

  • Improvement in motor function as measured by proportion of patients meeting the criteria for motor milestone responder using Hammersmith Infant Neurological Examination Section 2 (HINE-2)
  • Survival without the need for permanent ventilation

Safety: The most common side effects were lower respiratory infection (55%) and constipation (35%). Serious adverse reactions of atelectasis were more frequent in SPINRAZA-treated patients (18%) than in control patients (10%)

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Supportive trial: NURTURE6

Study: A phase 2, open-label, multicenter, multinational, single-arm trial

Treatment duration: Ongoing, results from a median of 2.9-year interim analysis

Participants: 25 presymptomatic, genetically diagnosed infants who were likely to develop Type 1 or 2 SMA aged ≤6 weeks at time of first dose

Primary endpoint: Survival without the need for permanent ventilation

Safety: Consistent with the SPINRAZA prescribing information

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

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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.

INDICATION

SPINRAZA is indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.

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. Biogen Data on File [as of 12/20]. 2. SPINRAZA. Prescribing Information. Cambridge, MA: Biogen; 2020. 3. Mercuri E, Darras BT, Chiriboga CA, et al. Nusinersen versus sham control in later-onset spinal muscular atrophy. N Eng J Med. 2018;378(7):625-635. 4. Darras BT, Chiriboga CA, Iannaccone ST, et al. Nusinersen in later-onset spinal muscular atrophy: long-term results from the phase 1/2 studies. Neurology. 2019;92(21):e2492-e2506. 5. Finkel RS, Mercuri E, Darras BT, et al. Nusinersen versus sham control in infantile-onset spinal muscular atrophy. N Eng J Med. 2017;377(18):1723-1732. 6. De Vivo DC, Bertini E, Swoboda KJ, et al. Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: interim efficacy and safety results from the Phase 2 NURTURE study. Neuromuscul Disord. 2019;29(11):842-856.

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