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Nusinersen Effective in Real-World SMA Treatment

Real-world, observational cohort data suggest that Biogen’s nusinersen (Spinraza) is safe and effective in the treatment of spinal muscular atrophy.

Tim Hagenacker, MD

New observational cohort data suggest that treatment with nusinersen (Spinraza; Biogen) can be administered safely, with efficacious results, in the real-world treatment of adults with 5q spinal muscular atrophy (SMA), the indication for which the therapy is FDA-approved.1

The study, which included 139 eligible screened patients, mean Hammersmith Functional Motor Scale Expanded (HFMSE) scores were increased from baseline by a mean difference of 1.73 (95% CI, 1.05—2.41; P <.0001) at 6 months, 2.58 (95% CI, 1.76—3.39; P <.0001) at 10 months, and 3.12 (95% CI, 2.06—4.19; P <.0001) at 14 months.

Overall, 89% (n = 124) of those eligible for analysis had 6-month data, 66% (n = 92) had 10-month data, and 41% (n = 57) had 14-month data. The analysis was conducted by Tim Hagenacker, MD, post-doctoral scientist and clinician, and group leader, Diseases of the Peripheral Nervous System Group, Neuroscience Lab, University Hospital Essen, and colleagues.

“Despite the limitations of the observational study design and a slow functional decline throughout the natural disease course, our data provide evidence for the safety and efficacy of nusinersen in the treatment of adults with 5q spinal muscular atrophy, with clinically meaningful improvements in motor function in a real-world cohort,” Hagenacker and colleagues wrote.

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Overall, there were clinically meaningful improvements, defined as a ≥3-point increase in HFMSE score, observed in 28% (n = 35) of 124 patients at 6 months, 35% (n = 33) of 92 patients at 10 months, and 40% (n = 23) of 57 patients at 14 months.

Through 14 months, the most frequent adverse events (AEs) among the whole of 173 patients screened were headache (35%; n = 61), back pain (22%; n = 38), and nausea (11%; n = 19). There were no serious AEs observed.

“Nusinersen is approved for the treatment of 5q spinal muscular atrophy of all types and stages in patients of all ages. Although clinical trials have shown improvements in motor function in infants and children treated with the drug, data for adults are scarce,” Hagenacker and colleagues detailed. “We aimed to assess the safety and efficacy of nusinersen in adults with 5q spinal muscular atrophy.”

They noted that future studies should aim to focus on the long-term effects, motor or motor-related functions like swallowing and ventilation, and if there is a need for individualization of treatment with dosing regimen and application intervals.

In late 2019, data from the SHINE trial (NCT02594124), an open-label extension study of nusinersen in patients with SMA suggested that the antisense oligonucleotide was associated to improvements or stabilization in ≥1 measures of motor function for up to nearly 6 years of treatment, in contrast to the expected decline observed in the natural history of the disease.2

That trial included 24 patients with SMA type 2 (n = 10) and type 3 (n = 14) who transitioned from the CS2/CS12 studies. Measures of motor function in the SHINE study analysis consisted of the HFMSE, the Upper Limb Module (ULM), and the 6-Minute Walk Test (6MWT). Notably, there were no discontinuations due to adverse events (AEs), and no additional safety concerns were identified during the almost 6 years of follow-up.


1. Hagenacker T, Wurster CD, Gunther R, et al. Nusinersen in adults with 5q spinal muscular atrophy: a non-interventional, multicentre, observational cohort study. Lancet Neurol. 2020;19(4):317-325. doi: 10.1016/S1474-4422(20)30037-5

2. Biogen Advances Spinal Muscular Atrophy (SMA) Clinical Research with New Study Evaluating a Higher Dose of SPINRAZA® (nusinersen) and Additional Data in a Broad Range of Patients [press release]. Cambridge, MA: Biogen; Published September 18, 2019. Accessed March 26, 2020. globenewswire.com/news-release/2019/09/18/1917265/0/en/Biogen-Advances-Spinal-Muscular-Atrophy-SMA-Clinical-Research-with-New-Study-Evaluating-a-Higher-Dose-of-SPINRAZA-nusinersen-and-Additional-Data-in-a-Broad-Range-of-Patients.html