Abstract Details

Title Effectiveness and Safety of Onasemnogene Abeparvovec in Older Patients with Spinal Muscular Atrophy (SMA): Real-World Outcomes from the RESTORE Registry

Topic Child Neurology and Developmental Neurology

Presentation(s) S39 - Child Neurology and Developmental Neurology (3:42 PM-3:54 PM)

Poster/Presentation
Number 002

Background

Interventional trials of OA demonstrated safety and efficacy in patients <6 months of age.

Objective We aimed to describe real-world outcomes in patients with SMA aged ≥6 months at the time of onasemnogene abeparvovec (OA) infusion.

Design/Methods We evaluated patients aged ≥6 months receiving OA (regardless of treatment with other disease-modifying therapies) in RESTORE (a comprehensive, prospective, multicenter, multinational, observational registry of patients with SMA).

Results As of May 23, 2021, RESTORE included 117 patients with SMA aged ≥6 months at OA infusion (51 [43.6%] were ≥6–<12 months; 57 [48.7%] were ≥12–<24 months; and 9 [7.7%] were ≥24 months). The majority of patients (n=73/117; 62.4%) had two copies of the SMN2 gene. Thirty-eight (32.5%) patients received OA monotherapy; 26 (22.2%) were administered OA that was preceded and followed by nusinersen and/or risdiplam; and 53 (45.3%) patients switched from nusinersen to OA. Of 81 patients with information available, 41 (50.6%) weighed ≥8.5 kg at OA infusion. At infusion, the majority of patients (n=76/114; 66.7%) were diagnosed with SMA type 1; 7/114 (6.1%) were presymptomatic. Of 28 patients with ≥2 evaluable CHOP INTEND assessments, 25 (89.3%) increased or maintained score, and 18 (64.3%) achieved ≥4-point increases (11 patients aged ≥6–<12 months; 7 patients aged ≥12–<24 months). Adverse event (AE) data were reported for 116 patients; 71 (61.2%) reported at least 1 treatment-emergent AE; 33 (28.4%) reported at least 1 serious AE (20 [17.2%] related to OA treatment).

Conclusions Patients with SMA aged ≥6 months at OA infusion benefited from treatment as measured by CHOP INTEND scores. The safety profile of OA in patients aged ≥6 months at infusion is consistent with the overall AE profile for OA, with no apparent pattern of AE incidence or severity according to age at infusion.

Authors/Disclosures
PRESENTER	No disclosure on file
Darryl C. De Vivo, MD, FAAN (Columbia University)	Dr. De Vivo has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen and Novartis. Dr. De Vivo has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Aspa Therapeutics.
Janbernd Kirschner	No disclosure on file
	No disclosure on file
Francesco Muntoni, MD (UCL Institute of Child Health)	Dr. Muntoni has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sarepta. Dr. Muntoni 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. Muntoni has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Sarepta. Dr. Muntoni has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Pfizer. Dr. Muntoni has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Biogen. Dr. Muntoni has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Avexis. Dr. Muntoni has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Roche. The institution of Dr. Muntoni has received research support from European Commission. The institution of Dr. Muntoni has received research support from Medical Research Council. The institution of Dr. Muntoni has received research support from Biogen. The institution of Dr. Muntoni has received research support from Muscular Dystrophy UK. The institution of Dr. Muntoni has received research support from MDA USA. The institution of Dr. Muntoni has received research support from Sarepta. The institution of Dr. Muntoni has received research support from Association Francoise Myopathies. Dr. Muntoni has received personal compensation in the range of $0-$499 for serving as a Clinical expert with UK NICE Committee.
	No disclosure on file
Susana Quijano-Roy (Aphp Paris Saclay, Hôpital Raymond Poincaré)	No disclosure on file
Kayoko Saito	No disclosure on file
	No disclosure on file
	No disclosure on file
Fred Anderson, PhD	Dr. Anderson has nothing to disclose.
Omar Dabbous	Omar Dabbous has received personal compensation for serving as an employee of Novartis Gene Therapies. Omar Dabbous has received stock or an ownership interest from Novartis Gene Therapies.
Richard S. Finkel, MD, FAAN (St. Jude Children's Research Hospital)	Dr. Finkel has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for AveXis. Dr. Finkel has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen. Dr. Finkel has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Catabasis. Dr. Finkel has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Capricor. Dr. Finkel has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for ReveraGen. Dr. Finkel 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. Dr. Finkel 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. Finkel has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Elsevier. The institution of Dr. Finkel has received research support from AveXis. The institution of Dr. Finkel has received research support from Biogen. The institution of Dr. Finkel has received research support from Capricor. The institution of Dr. Finkel has received research support from Catabasis. The institution of Dr. Finkel has received research support from ReveraGen. The institution of Dr. Finkel has received research support from Roche. The institution of Dr. Finkel has received research support from Scholar Rock. Dr. Finkel has received intellectual property interests from a discovery or technology relating to health care. An immediate family member of Dr. Finkel has received intellectual property interests from a discovery or technology relating to health care. Dr. Finkel has received personal compensation in the range of $0-$499 for serving as a Speaker in workshop with National Academy of Sciences. Dr. Finkel has a non-compensated relationship as a advisor with n-Lorem Foundation that is relevant to AAN interests or activities. Dr. Finkel has a non-compensated relationship as a Board Member with EveryLife Foundation that is relevant to AAN interests or activities.