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Open Access 09-05-2024 | Spinal Muscular Atrophy | Original Research

A Post-Marketing Surveillance Study of Nusinersen for Spinal Muscular Atrophy in Routine Medical Practice in China: Interim Results

Authors: Yuwu Jiang, Yi Wang, Hui Xiong, Wenhui Li, Rong Luo, Wenxiong Chen, Fei Yin, Junlan Lü, Jianmin Liang, Wan-Jin Chen, Xinguo Lu, Hua Wang, Jihong Tang, Michael Monine, Corinne Makepeace, Xin Jin, Richard Foster, Russell Chin, Zdenek Berger

Published in: Advances in Therapy | Issue 7/2024

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Abstract

Introduction

Spinal muscular atrophy (SMA) is a rare, autosomal recessive, neuromuscular disease that leads to progressive muscular weakness and atrophy. Nusinersen, an antisense oligonucleotide, was approved for SMA in China in February 2019. We report interim results from a post-marketing surveillance phase 4 study, PANDA (NCT04419233), that collects data on the safety, efficacy, and pharmacokinetics of nusinersen in children with SMA in routine clinical practice in China.

Methods

Participants enrolled in PANDA will be observed for 2 years following nusinersen treatment initiation. The primary endpoint is the incidence of adverse events (AEs)/serious AEs (SAEs) during the treatment period. Efficacy assessments include World Health Organization (WHO) Motor Milestones assessment, the Hammersmith Infant Neurological Examination (HINE), and ventilation support. Plasma and cerebrospinal fluid (CSF) concentrations of nusinersen are measured at each dose visit.

Results

Fifty participants were enrolled as of the January 4, 2023, data cutoff: 10 with infantile-onset (≤ 6 months) and 40 with later-onset (> 6 months) SMA. All 50 participants have received at least one dose of nusinersen; 6 have completed the study. AEs were experienced by 45 (90%) participants and were mostly mild/moderate; no AEs led to nusinersen discontinuation or study withdrawal. Eleven participants experienced SAEs, most commonly pneumonia (n = 9); none were considered related to study treatment. Stability or gain of WHO motor milestone was observed and mean HINE-2 scores improved in both subgroups throughout the study. No serious respiratory events occurred, and no permanent ventilation support was initiated during the study. Pre-dose nusinersen CSF concentrations increased steadily through the loading-dose period, with no accumulation in plasma after multiple doses.

Conclusion

Nusinersen was generally well tolerated with an acceptable overall safety profile, consistent with the known safety of nusinersen. Efficacy, safety, and nusinersen exposure are consistent with prior observations. These results support continuing PANDA and evaluation of nusinersen in Chinese participants with SMA.

Trial Registration

ClinicalTrials.gov identifier, NCT04419233.
Appendix
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Literature
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go back to reference Darras BT, Monani UR, De Vivo DC. Genetic disorders affecting the motor neuron: spinal muscular atrophy. Swaiman’s Pediatric Neurology. 6th ed. Edinburgh: Elsevier; 2017. Darras BT, Monani UR, De Vivo DC. Genetic disorders affecting the motor neuron: spinal muscular atrophy. Swaiman’s Pediatric Neurology. 6th ed. Edinburgh: Elsevier; 2017.
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go back to reference Tulinius M, Castro D, Finkel RS, et al. Long-term safety and efficacy of nusinersen in infantile-onset spinal muscular atrophy: 5-year interim from SHINE. SMA-Europe - 3rd International Scientific Congress on Spinal Muscular Atrophy; Barcelona, Spain; 2022. Tulinius M, Castro D, Finkel RS, et al. Long-term safety and efficacy of nusinersen in infantile-onset spinal muscular atrophy: 5-year interim from SHINE. SMA-Europe - 3rd International Scientific Congress on Spinal Muscular Atrophy; Barcelona, Spain; 2022.
Metadata
Title
A Post-Marketing Surveillance Study of Nusinersen for Spinal Muscular Atrophy in Routine Medical Practice in China: Interim Results
Authors
Yuwu Jiang
Yi Wang
Hui Xiong
Wenhui Li
Rong Luo
Wenxiong Chen
Fei Yin
Junlan Lü
Jianmin Liang
Wan-Jin Chen
Xinguo Lu
Hua Wang
Jihong Tang
Michael Monine
Corinne Makepeace
Xin Jin
Richard Foster
Russell Chin
Zdenek Berger
Publication date
09-05-2024
Publisher
Springer Healthcare
Published in
Advances in Therapy / Issue 7/2024
Print ISSN: 0741-238X
Electronic ISSN: 1865-8652
DOI
https://doi.org/10.1007/s12325-024-02852-7

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