Nusinersen Medicine 04 December 2025 Extended indication Nusinersen monotherapy for treatment of 5q Spinal muscular atrophy in newborns, infants, toddlers, children, adolescents and adults administered at a higher dose Therapeutic value Possible added value Total cost Registration phase Registration application pending Product Active substance Nusinersen Domain Neurological disorders Reason of inclusion Indication extension IND Main indication SMA Extended indication Nusinersen monotherapy for treatment of 5q Spinal muscular atrophy in newborns, infants, toddlers, children, adolescents and adults administered at a higher dose Current proprietary name Already available biosimilars / generics Proprietary name Spinraza Manufacturer Biogen Portfolio holder Biogen Mechanism of action Antisense oligonucleotide Route of administration Intrathecal Therapeutical formulation Injection Budgetting framework Centre of expertise UMC Utrecht Additional remarks Registration Registration route Type of trajectory Particularity ATMP No Submission date January 2025 Expected Registration February 2026 Orphan drug Yes Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Zolgensma, Evrysdi, Spinraza Therapeutic value Possible added value This assessment does not indicate any potential inclusion in the package. Substantiation The higher dose cohort showed statistically significant improvement over the matched sham comparator on the primary endpoint of change in CHOP-INTEND from baseline to six months (least squares mean difference: 26.19; p<0.0001). Results favored the higher dose regimen relative to sham across secondary endpoints and trended in favor of the higher dose regimen over the currently approved 12mg regimen on key biomarker and efficacy measures. The higher dose regimen was generally well tolerated, with reported adverse events generally consistent with SMA and the known safety profile of nusinersen. The percentage of serious adverse events was lower in the higher dose regimen (60%) as compared to the 12 mg group (72%(. Duration of treatment Frequency of administration 1 times every 4 months Dosage per administration 28 mg References 1. https://investors.biogen.com/news-releases/news-release-details/biogen-announces-positive-topline-results-study-higher-dose 2. ClinicalTrials.gov. Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy (DEVOTE). NCT04089566. https://clinicaltrials.gov/study/NCT04089566 Additional remarks Expected patient volume per year Patient volume Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Additional remarks Expected cost per patient per year Cost References Additional remarks Potential total cost per year Total cost Total cost for sluice Additional remarks Off label use Off label use No Indications off label use References Additional remarks Indication extension Indication extensions Yes Indication extensions Nusinersen monotherapy for treatment of 5q Spinal muscular atrophy in newborns, infants, toddlers, children, adolescents and adults administered at a higher dose References Additional remarks Other information Additional remarks