WHY SPINRAZA/EARLY-ONSET EFFICACY

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

Pivotal trial: ENDEAR1,2

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: Survival without the need for permanent ventilation and proportion of patients meeting the criteria for motor milestone responder using HINE-2

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%)

SEE ADDITIONAL SPINRAZA SAFETY

Better event-free survival compared to untreated patients. Event-free survival was defined as the primary endpoint, no death or use of permanent assisted ventilation2

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

MEASURES OF MOBILITY TOOL

Those with Type 1 SMA saw improvements in motor milestones that are rarely,
if ever, achieved in untreated children2

51% treated vs 0% untreated achieved the motor milestone responder criteria as defined by HINE-2 at 13 months.† Motor milestones included2:

†P<0.00012
A treatment responder was defined as any patient achieving an improvement in more categories of motor milestones than worsening according to HINE-2.1
HINE-2=Hammersmith Infant Neurological Examination Section 2, motor milestone portion.

Patients treated with SPINRAZA continued to improve over time, as measured at day 394, while untreated patients saw a decline in motor milestones2

The ENDEAR Trial concluded early because of the results of the interim analysis, hence why not all patients were assessed at Day 394.

SEM=standard error of the mean.

a child with early-onset SMA who is being treated with SPINRAZA

“Whether she can or can’t do something, she is going to try.” —Mother of Sofia

Review the warnings and precautions, including thrombocytopenia, coagulation abnormalities,
and renal toxicity1

SEE SPINRAZA SAFETY
Start your patient on SPINRAZA

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.

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

References

1. SPINRAZA. Prescribing Information. Biogen; 2023. 2. Finkel RS, Mercuri E, Darras BT, et al; ENDEAR Study Group. Nusinersen versus sham control in infantile-onset spinal muscular atrophy. N Engl J Med. 2017;377(18):1723-1732.

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