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Why SPINRAZA/Presymptomatic Efficacy

SPINRAZA has proven efficacy in presymptomatic, early-, and later-onset SMA1-4

Supportive trial: NURTURE1

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

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: Time to death or respiratory intervention

Secondary endpoints: Attainment of WHO motor milestones and change from baseline in the CHOP INTEND motor function scale

Study limitations: Small participant group size and no sham control group; CHOP INTEND score was assessed only until a participant achieved the maximum score of 64. HINE-2 score was assessed until day 778, but not at later study visits

Safety: Consistent with the SPINRAZA prescribing information; no new safety concerns were identified. 80% (20/25) of participants had an adverse event (AE) that was mild or moderate in severity. Eight participants experienced AEs that were possibly or definitely related to the lumbar puncture procedure.

SEE ADDITIONAL SPINRAZA SAFETY

All the following are results after a median follow-up of 2.9 years in the study1

100% (25/25) of presymptomatic infants were alive without permanent ventilation1

The majority of infants achieved the following WHO motor milestones:

The natural history of SMA shows infants with Type 1 would not survive or would require permanent ventilation by 13.5 months of age1

100% (25/25) of presymptomatic infants treated with SPINRAZA continued to gain motor skills over a median of 2.9 years of follow-up1

Mean CHOP INTEND scores showed SPINRAZA improved motor function, with scores rising steadily until day 183. The majority of patients achieved a maximum score of 64 and then remained stable over time.

CHOP INTEND=Children’s Hospital of Philadelphia Infant Test of Neuromuscular Disorders.
SE=standard error.

**In the original protocol, CHOP INTEND was to be assessed in participants at each visit up to day 778; however, this was amended to be until they had a maximum score of 64. Once a score of 64 was achieved, CHOP INTEND was no longer assessed.

Learn more about the mobility measures used in the SPINRAZA clinical trials.

MEASURES OF MOBILITY TOOL
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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. 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. 2. Finkel RS, Mercuri E, Darras BT, et al. Nusinersen versus sham control in infantile-onset spinal muscular atrophy. N Engl J Med. 2017;377(18):1723-1732. 3. Mercuri E, Darras BT, Chiriboga CA, et al. Nusinersen versus sham control in later-onset spinal muscular atrophy. N Engl 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.

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