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. 2024 Jan:34:41-48.
doi: 10.1016/j.nmd.2023.11.010. Epub 2023 Dec 2.

Continued safety and long-term effectiveness of onasemnogene abeparvovec in Ohio

Megan A Waldrop  1 , Shannon Chagat  2 , Michael Storey  3 , Alayne Meyer  4 , Megan Iammarino  4 , Natalie Reash  4 , Lindsay Alfano  4 , Linda Lowes  4 , Garey Noritz  5 , Andre Prochoroff  6 , Ian Rossman  7 , Matthew Ginsberg  7 , Kathryn Mosher  8 , Eileen Broomall  9 , Nancy Bass  10 , Courtney Gushue  11 , Kavitha Kotha  11 , Grace Paul  11 , Richard Shell  11 , Chang-Yong Tsao  12 , Jerry R Mendell  13 , Anne M Connolly  13
Affiliations

Continued safety and long-term effectiveness of onasemnogene abeparvovec in Ohio

Megan A Waldrop et al. Neuromuscul Disord. 2024 Jan.

Abstract

5q spinal muscular atrophy (SMA) is an autosomal recessive neurodegenerative disease caused by absence of the SMN1 gene with three FDA approved genetic therapies which significantly improve outcomes. The AAV9 mediated gene replacement therapy, onasemnogene abeparvovec, has the greatest potential for side effects. Here we report the safety and outcomes from 46 children treated with onasemnogene abeparvovec in the state of Ohio between December 2018 and January 2023. In our cohort, onasemnogene abeparvovec treatment remained safe and no child experienced any significant adverse events, including thrombotic microangiopathy, liver failure or death. All children experienced benefit, although the benefit in those with 2 copies of SMN2 was variable. 79 % of the children treated when symptomatic had a SMN2 modifying therapy added on. With careful screening and post treatment monitoring, onasemnogene abeparvovec is safe and effective for children with SMA in the state of Ohio, but more work needs to be done to ensure optimal outcomes for all children with 2 copies of SMN2.

Keywords: Gene therapy; Onasemnogene abeparvovec; Spinal muscular atrophy.

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Conflict of interest statement

Declaration of Competing Interest Dr. Waldrop has served on advisory boards for Novartis Gene Therapies and received clinical trial support for Novartis Gene Therapies. Dr. Storey has served on an advisory board for and received consulting fees from Novartis Gene Therapies. Lindsay Alfano has served on an advisory board from Genentech-Roche. Dr. Lowes has received grant support from Novartis Gene Therapies. Dr. Bass has served on speaker and advisory boards for Biogen. Dr. Shell has served as a consultant for Novartis Gene Therapies and serves on an adjudication committee for Biogen. Dr. Mendell has received clinical trial support and personal fees from Novartis Gene Therapies. Dr. Connolly has served on advisory boards for Biohaven and Scholar Rock. All other authors have no conflicts of interest relevant to this article to disclose.

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