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Abstract Details

SUNFISH Parts 1 and 2: 4-year Efficacy and Safety Data of Risdiplam in Types 2 and 3 Spinal Muscular Atrophy (SMA)
Child Neurology and Developmental Neurology
S34 - Child Neurology and Developmental Neurology 2 (2:36 PM-2:48 PM)
009

SMA affects individuals with a broad age range and spectrum of disease severity. Risdiplam is a centrally and peripherally distributed, oral survival of motor neuron 2 (SMN2) pre-mRNA splicing modifier that has been approved in over 90 countries worldwide.

To determine the efficacy and safety of risdiplam (EVRYSDI®) in patients with Types 2 and 3 SMA after 48 months of treatment.

SUNFISH (NCT02908685) is a multicenter, two-part, randomized, placebo-controlled, double-blind study in patients with Types 2 and 3 SMA (inclusion criteria: aged 2–25 years at enrollment). Part 1 (N=51) assessed the safety, tolerability and pharmacokinetics/pharmacodynamics of different risdiplam dose levels in patients with Types 2 and 3 SMA (ambulant and non-ambulant). 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. In Part 2, participants were treated with risdiplam or placebo for 12 months; participants 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.

The primary endpoint (Part 2) of change from baseline in the 32-item Motor Function Measure (MFM32) total score in risdiplam (n=120) versus placebo (n=60) was met at Month 12. These increases in motor function were sustained in the second and third year after risdiplam treatment, as measured by changes in MFM32, Hammersmith Functional Motor Scale – Expanded, and Revised Upper Limb Module. At Month 36 (data-cut: 6 September 2021), there were no treatment-related safety findings leading to withdrawal from either SUNFISH Part 1 or 2. Here we present 48-month efficacy and safety data from SUNFISH.

SUNFISH is ongoing and will provide further long-term efficacy and safety data of risdiplam in a broad population of children, teenagers and adults with SMA.

Authors/Disclosures
Laurent Servais (John Radcliffe Hospital)
PRESENTER
Laurent Servais has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Biogen. Laurent Servais has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Roche. Laurent Servais has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Novartis. Laurent Servais has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Astellas. Laurent Servais has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Pfizer. Laurent Servais has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sarepta. The institution of Laurent Servais has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Evox. Laurent Servais has received personal compensation in the range of $500-$4,999 for serving as a Consultant for PTC. Laurent Servais has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Sysnav. Laurent Servais has received personal compensation in the range of $500-$4,999 for serving as a Consultant for BioHaven. Laurent Servais has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Dyne. Laurent Servais has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Zentech. Laurent Servais has received personal compensation in the range of $500-$4,999 for serving as a Consultant for MitoRx. Laurent Servais has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Lupin. Laurent Servais has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Fibrogen. Laurent Servais has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alltrana. Laurent Servais has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Illumina. Laurent Servais has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche. The institution of Laurent Servais has received research support from Roche. The institution of Laurent Servais has received research support from Novartis. The institution of Laurent Servais has received research support from Biogen. The institution of Laurent Servais has received research support from Zentech. The institution of Laurent Servais has received research support from BioHaven. The institution of Laurent Servais has received research support from PerkinHalmers. The institution of Laurent Servais has received research support from Scholar Rock.
Maryam Oskoui, MD, FAAN (Montreal Children's Hospital - McGill University Health Centre) Dr. Oskoui has received personal compensation in the range of $500-$4,999 for serving as an officer or member of the Board of Directors for the Association des Neurologues du Quebec. The institution of Dr. Oskoui has received research support from Biogen. The institution of Dr. Oskoui has received research support from Roche Genetech. The institution of Dr. Oskoui has received research support from Muscular Dystrophy Canada. The institution of Dr. Oskoui has received research support from Canadian Institutes of Health Research. Dr. Oskoui has a non-compensated relationship as a Member of the Medical and Scientific Advisory Committee with Muscular Dystrophy Canada that is relevant to AAN interests or activities. Dr. Oskoui has a non-compensated relationship as a Member of the External Advisory Board with MDUK Oxford Neuromuscular Centre that is relevant to AAN interests or activities.
John W. Day, MD, PhD (Stanford University School of Medicine) Dr. Day has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Affinia Therapeutics. Dr. Day has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis Gene Therapy. Dr. Day has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen. Dr. Day has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche/Genentech. Dr. Day has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sarepta. Dr. Day has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Scholar Rock. Dr. Day has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Avidity. Dr. Day has received personal compensation in the range of $10,000-$49,999 for serving as an officer or member of the Board of Directors for Muscular Dystrophy Association. The institution of Dr. Day has received research support from Audentes. The institution of Dr. Day has received research support from Novartis Gene Therapy. The institution of Dr. Day has received research support from Biogen. The institution of Dr. Day has received research support from Cytokinetics. The institution of Dr. Day has received research support from Roche/Genentech. The institution of Dr. Day has received research support from Sanofi/Genzyme. The institution of Dr. Day has received research support from Sarepta. The institution of Dr. Day has received research support from Scholar Rock. Dr. Day has received intellectual property interests from a discovery or technology relating to health care.
Nicholas Deconinck Nicholas Deconinck has received personal compensation in the range of $500-$4,999 for serving as a Consultant for biogen. Nicholas Deconinck has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for roche. Nicholas Deconinck has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for avexis. Nicholas Deconinck has received publishing royalties from a publication relating to health care.
Elena Mazzone Elena Mazzone has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Avexis. Elena Mazzone has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Roche. Elena Mazzone has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant for Biogen.
Andrés Nascimento, MD (Hospital Sant Joan de Déu) Dr. Nascimento has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche, Novartis , Biogen. Dr. Nascimento has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Roche, Novartis, Biogen.
Kayoko Saito, MD, PhD Dr. Saito has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis Gene Therapies, Inc./Novartis. Dr. Saito has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen. Dr. Saito has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Chugai (Roche). Dr. Saito has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Novartis Gene Therapies, Inc./Novartis. Dr. Saito has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Chugai (Roche). The institution of Dr. Saito has received research support from Biogen.
Carole Vuillerot Carole Vuillerot has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Hoffmann Roche. Carole Vuillerot has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen. Carole Vuillerot has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen . Carole Vuillerot has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche Hoffmann. Carole Vuillerot has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Avexis.
Giovanni Baranello Giovanni Baranello has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Roche. Giovanni Baranello has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Roche. Giovanni Baranello has received personal compensation in the range of $500-$4,999 for serving as a Consultant for AveXis. Giovanni Baranello has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche. Giovanni Baranello has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for AveXis. Giovanni Baranello has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Roche. Giovanni Baranello has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for AveXis. The institution of Giovanni Baranello has received research support from Great Ormond Street Hospital Charity.
Odile Boespflug-Tanguy Odile Boespflug-Tanguy has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Minoryx. Odile Boespflug-Tanguy has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Inventiva. Odile Boespflug-Tanguy has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for BLUEBIRD.
Nathalie M. Goemans, MD (University Hospitals Leuven) Dr. Goemans has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Pfizer. Dr. Goemans has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Biogen. Dr. Goemans has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Roche. Dr. Goemans has received publishing royalties from a publication relating to health care.
Janbernd Kirschner Janbernd Kirschner has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Roche. Janbernd Kirschner has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Novartis. Janbernd Kirschner has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Pfizer. Janbernd Kirschner has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen. Janbernd Kirschner has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Genethon. Janbernd Kirschner has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Italfarmaco. Janbernd Kirschner has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Pfizer. Janbernd Kirschner has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for PTC. The institution of Janbernd Kirschner has received research support from Roche. The institution of Janbernd Kirschner has received research support from Biogen. The institution of Janbernd Kirschner has received research support from Novartis.
Anna *USE ID 361430 Kostera-Pruszczyk Anna Kostera-Pruszczyk has nothing to disclose.
Jessica Braid, Other (Roche Products Ltd) Miss Braid has received stock or an ownership interest from Roche Products Ltd.
Gergely Zoltan Papp, MD (F. Hoffmann-La Roche AG) Dr. Papp has received personal compensation for serving as an employee of F. Hoffmann-La Roche AG.
Ksenija Gorni Dr. Gorni has received personal compensation for serving as an employee of Hoffman La Roche.
Carmen Martin Carmen Martin has received personal compensation for serving as an employee of Roche Products Ltd. Carmen Martin has received stock or an ownership interest from F. Hoffmann-La Roche Ltd.
Renata Scalco (Roche) Renata SCALCO has received personal compensation for serving as an employee of Roche. Renata SCALCO has received stock or an ownership interest from Roche.
Wai Yin Yeung Wai Yin Yeung has nothing to disclose.
Eugenio Mercuri Eugenio Mercuri has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sarepta, Santhera, PTC, Pfizer, Roche, Biogen, Avexis Novartis, Scholar Rock, Cytokinetics. Eugenio Mercuri has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Sarepta, Santhera, PTC, Pfizer, Roche, Biogen, Avexis Novartis. The institution of Eugenio Mercuri has received research support from biogen.