PMID- 34809526
OWN - NLM
STAT- MEDLINE
DCOM- 20230706
LR  - 20230706
IS  - 1563-5279 (Electronic)
IS  - 0020-7454 (Linking)
VI  - 133
IP  - 8
DP  - 2023 Dec
TI  - Real-world safety of nusinersen in Japan: results from an interim analysis of a 
      post-marketing surveillance and safety database.
PG  - 851-863
LID - 10.1080/00207454.2021.1995382 [doi]
AB  - PURPOSE: Nusinersen is the first disease-modifying therapy to treat spinal 
      muscular atrophy (SMA). This report describes the safety and effectiveness of 
      nusinersen in Japanese clinical use using two data sources: an ongoing Japanese 
      post-marketing surveillance (PMS) and the safety database of the marketing 
      authorisation holder, Biogen . MATERIALS AND METHODS: The PMS is evaluating the 
      safety and effectiveness of nusinersen in all patients treated with nusinersen in 
      Japan between August 2017 and August 2025; this interim analysis included data up 
      to May 30, 2019. Biogen safety database data up to June 30, 2019 were also 
      included to capture adverse events (AEs) from after the interim analysis cutoff 
      date. Collected data included medical history, dosage and administration, and 
      AEs. Safety assessment included AEs and serious AEs (SAEs). Effectiveness 
      analyses included motor function assessments and clinical global impressions of 
      improvement. RESULTS: Of 271 patients in the PMS population, 94 had SMA type I 
      (34.7%), and 177 had SMA types II-IV (65.3%). AEs occurred in 67 patients (24.7%) 
      and SAEs in 23 patients (8.5%). The Biogen safety database contained reports of 
      345 AEs; the most common were pneumonia, headache, and pyrexia, consistent with 
      symptoms of SMA and lumbar puncture. In the analysis set, 26.2% of patients 
      receiving nusinersen showed motor function improvements and 99.6-100.0% showed 
      overall improvement. CONCLUSION: In this interim analysis of the PMS and Biogen 
      safety database, nusinersen had a favourable benefit-risk profile in Japanese 
      patients with SMA.
FAU - Wataya, Takafumi
AU  - Wataya T
AD  - Biogen Japan, Tokyo, Japan.
FAU - Takasaki, Sakura
AU  - Takasaki S
AD  - Biogen Japan, Tokyo, Japan.
FAU - Hoshino, Misuzu
AU  - Hoshino M
AD  - Biogen Japan, Tokyo, Japan.
FAU - Makioka, Haruki
AU  - Makioka H
AD  - Biogen Japan, Tokyo, Japan.
FAU - Nakamura, Genshu
AU  - Nakamura G
AD  - Biogen Japan, Tokyo, Japan.
FAU - Matsuda, Naoto
AU  - Matsuda N
AUID- ORCID: 0000-0002-1855-682X
AD  - Biogen Japan, Tokyo, Japan.
LA  - eng
PT  - Journal Article
DEP - 20211122
PL  - England
TA  - Int J Neurosci
JT  - The International journal of neuroscience
JID - 0270707
RN  - 5Z9SP3X666 (nusinersen)
RN  - 0 (Oligonucleotides)
SB  - IM
MH  - Humans
MH  - Japan
MH  - Oligonucleotides/adverse effects
MH  - *Spinal Muscular Atrophies of Childhood/drug therapy
MH  - *Muscular Atrophy, Spinal/drug therapy
MH  - Marketing
MH  - Product Surveillance, Postmarketing
OTO - NOTNLM
OT  - Benefit-risk profile
OT  - drug safety
OT  - nusinersen
OT  - post-marketing
OT  - spinalmuscular atrophy
OT  - surveillance
EDAT- 2021/11/24 06:00
MHDA- 2023/07/06 06:42
CRDT- 2021/11/23 05:30
PHST- 2023/07/06 06:42 [medline]
PHST- 2021/11/24 06:00 [pubmed]
PHST- 2021/11/23 05:30 [entrez]
AID - 10.1080/00207454.2021.1995382 [doi]
PST - ppublish
SO  - Int J Neurosci. 2023 Dec;133(8):851-863. doi: 10.1080/00207454.2021.1995382. Epub 
      2021 Nov 22.