.

WHY SPINRAZA/LATER-ONSET EFFICACY

Extensive data substantiate the clinical benefit of SPINRAZA in presymptomatic, early-, and
later-onset SMA.1-4

Pivotal trial: CHERISH3,5

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

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 HFMSE

Secondary endpoint: Change in upper limb function as measured by RULM

Study limitations: Differences in dosing compared to the approved SPINRAZA schedule

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

SEE ADDITIONAL SPINRAZA SAFETY

Individuals treated with SPINRAZA experienced improvements in motor and upper limb function over 15 months.3

Those with later-onset SMA treated with SPINRAZA experienced a 3.9 improvement in motor function

HFMSE=Hammersmith Functional Motor Scale—Expanded.

Motor function began to steadily improve in just 6 months compared to untreated group.3

those with later-onset SMA treated with SPINRAZA experienced a 4.2 improvement in upper limb function

RULM=The Revised Upper Limb Module evaluates limb function and strength in individuals with later-onset SMA who are unable to walk. It is scored from 0-37 points, with higher scores indicating better function.

Supportive trials: CS2/CS124

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

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

Study limitations: Differences in dosing compared to the approved SPINRAZA schedule and no control group

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

SEE ADDITIONAL SPINRAZA SAFETY

Individuals treated with SPINRAZA saw increases in upper limb and motor function over 3 years.4

HFMSE was developed specifically for SMA and is a widely used and well-validated scale for measuring clinical benefit in later-onset SMA.6,7

MEASURES OF MOBILITY

ULM=The Upper Limb Module evaluates limb function and strength in individuals with later-onset SMA who are unable to walk. It is scored from 0-18 points, with higher scores indicating better function.

100% (7/7) who achieved meaningful improvements in their walking distance maintained these milestones through
year 3.8

those with Type 3 later-onset SMA treated with SPINRAZA increased their walking distance by 396.7 meters

SPINRAZA may help patients gain or regain the ability to walk.4

  • At least 1 of 11 patients with Type 2 SMA gained the ability to walk for the first time
  • At least 2 of 4 patients with Type 3 SMA regained the ability to walk
Ian Quote

“My belief is that anyone can do anything. It doesn’t matter if you’re in a wheelchair, it doesn’t take much strength to make
a big impact.”

–Byron

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

SEE SPINRAZA SAFETY
Start your patient on SPINRAZA

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):1723-173 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. 5. SPINRAZA [prescribing information]. Cambridge, MA: Biogen; 2020. 6. Finkel RA, Bertini EB, Muntoni FC, et al. 209th ENMC International Workshop: Outcome Measures and Clinical Trial Readiness in Spinal Muscular Atrophy 7–9 November 2014, Heemskerk, The Netherlands. Neuromuscul Disord. 2015;25:593-602. 7. O’Hagen JM, Glanzman AM, Michael PM, et al. An expanded version of the Hammersmith Functional Motor Scale for SMA II and III patients. Neuromuscul Disord. 2007;17:693-697. 8. Biogen Data on File [as of 12/19].

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