In:
Journal of Neurology, Neurosurgery & Psychiatry, BMJ, Vol. 93, No. 6 ( 2022-06), p. A95.3-A95
Abstract:
SUNFISH ( NCT02908685 ) is a multicentre, two-part, randomised (2:1, risdiplam:placebo), placebo-con- trolled, double-blind study in individuals with Type 2/3 spinal muscular atrophy (SMA; inclusion criteria 2–25 years at enrolment). SUNFISH investigates efficacy and safety of risdiplam, a centrally and periph- erally distributed oral survival of motor neuron 2 pre-mRNA splicing modifier. Risdiplam (EVRYSDI™) has been approved by the US Food and Drug Administration for the treatment of individuals with SMA, aged 2 months and older. In Part 1 (N=51) the safety, tolerability and pharmacokinetics/pharmacodynamics of different risdiplam dose levels were assessed. Part 2 (N=180) assessed the efficacy and safety of the Part 1-selected dose of risdiplam versus placebo in Type 2 and non-ambulant Type 3 SMA. Individuals received risdiplam or placebo for 12 months; all individuals then received risdiplam until Month 24, when they had the oppor- tunity to enter the open-label extension phase. In Part 2, the primary outcome of the study was met, showing a statistically significant difference in change from baseline in 32-item Motor Function Measure total score at Month 12 between individuals treated with risdiplam (n=120) and placebo (n=60). No treatment-related safety findings leading to withdrawal were reported. Here we present SUNFISH Part 2 data after 24 months’ treatment. g.baranello@ucl.ac.uk 16
Type of Medium:
Online Resource
ISSN:
0022-3050
,
1468-330X
DOI:
10.1136/jnnp-2022-ABN.309
Language:
English
Publisher:
BMJ
Publication Date:
2022
detail.hit.zdb_id:
1480429-3
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