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C.2 SUNFISH parts 1 and 2: 4-year efficacy and safety data of risdiplam in types 2 and 3 spinal muscular atrophy (SMA)

Published online by Cambridge University Press:  05 June 2023

JK Mah
Affiliation:
(Calgary)*
M Oskoui
Affiliation:
(Montreal)
JW Day
Affiliation:
(Palo Alto)
N Deconinck
Affiliation:
(Brussles)
E Mazzone
Affiliation:
(Rome)
A Nascimento
Affiliation:
(Barcelona)
K Saito
Affiliation:
(Tokyo)
C Vuillerot
Affiliation:
(Lyon)
G Baranello
Affiliation:
(London)
O Boespflug-Tanguy
Affiliation:
(Paris)
N Goemans
Affiliation:
(Leuven)
J Kirschner
Affiliation:
(Freiburg)
A Kostera-Pruszczyk
Affiliation:
(Warsaw)
L Servais
Affiliation:
(Oxford)
J Braid
Affiliation:
(Welwyn Garden City)
M Gerber
Affiliation:
(Basel)
K Gorni
Affiliation:
(Basel)
C Martin
Affiliation:
(Welwyn Garden City)
W Yeung
Affiliation:
(Welwyn Garden City)
RS Scalco
Affiliation:
(Basel)
E Mercuri
Affiliation:
(Rome)
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Abstract

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Background: SMA affects individuals with a broad age range and spectrum of disease severity. Risdiplam (EVRYSDI®) is a centrally and peripherally distributed, oral SMN2 pre-mRNA splicing modifier. Methods: SUNFISH is a multicenter, two-part, randomized, placebo-controlled, double-blind study in patients with Types 2/3 SMA. Part 1 assessed the safety, tolerability and pharmacokinetics/pharmacodynamics of different risdiplam dose levels in patients with Types 2/3 SMA. Part 2 assessed the efficacy and safety of the selected dose of risdiplam versus placebo in Type 2 and non-ambulant Type 3 SMA. In Part 2, participants were treated with risdiplam or placebo for 12 months, then received risdiplam in a blinded manner until month 24. At month 24, patients were offered the opportunity to enter the open-label extension phase. Results: Change from baseline in MFM32 total score (Part 2- primary endpoint) in patients treated with risdiplam versus placebo was met at month 12. These increases in motor function were sustained in the second and third year after risdiplam treatment. Here we present 4-year efficacy and safety data from SUNFISH. Conclusions: SUNFISH is ongoing and will provide further long-term efficacy and safety data of risdiplam in a broad population of individuals with SMA.

Type
Abstracts
Copyright
© The Author(s), 2023. Published by Cambridge University Press on behalf of Canadian Neurological Sciences Federation