Delphi consensus on gene therapy of spinal muscular atrophy with onasemnogene abeparvovec in Germany, Austria and Switzerland-part I-systematic literature review and existing evidence

Weiß C, Vill K, Baumann M, Bernert G, Blaschek A, Eisenkölbl A, Flotats-Bastardas M, Friese J, Ganter C, Goldhahn K, Hahn A, von der Hagen M, Hartmann H, Hasselmann O, Horber V, Husain RA, Illsinger S, Jacquier D, Johannsen J, Köhler C, Kölbel H, Kolodzig M, Klein A, Pechmann A, von Moers A, Müller-Felber W, Rauscher C, Schara-Schmidt U, Schreiber G, Schwartz O, Sproß J, Stettner GM, Stoltenburg C, Stumpe E, Trollmann R, Wiegand G, Wilichowski E, Kirschner J, Ziegler A (2026)

Publication Type: Journal article

Publication year: 2026

Journal

Book Volume: 13

Pages Range: 420-435

Journal Issue: 3

DOI: 10.1177/22143602251387019

Abstract

BackgroundSince the approval of onasemnogen abeparvovec (OA) for gene addition therapy in children with spinal muscular atrophy (SMA), there has been a considerable increase of evidence regarding its effectiveness and safety. Consequently, the previous recommendations needed to be revised.ObjectiveThe primary objective was to develop an evidence- and expert-based best practice protocol ensuring optimal patient safety and comprehensive support for affected families. The harmonization of treatment algorithms is expected to facilitate the collection of standardized real-world data, laying the foundation for future evidence-based adjustments.MethodsA modified, two-part Delphi process was selected as a standardized methodology. Experts specializing in SMA from all 31 neuromuscular treatment centers within Germany, Austria and Switzerland, and patient advocacy groups participated in an industry-independent Delphi panel. Existing evidence concerning effectiveness, safety, and guidelines of OA was analyzed in a systematic literature followed by development of consensus statements regarding its effectiveness.ResultsStrong consensus was reached regarding the following statements on effectiveness: (1) OA gene addition therapy for SMA demonstrates a clear advantage compared to the natural progression of the disease. (2) Superiority of any of the three approved disease-modifying therapies has not been proven. (3) Earlier initiation of therapy with fewer symptoms and shorter disease duration leads to better outcomes. (4) There is no clinical evidence supporting the superiority of combining two treatments over monotherapy.Conclusions: The systematic literature analysis constitutes the basis for the subsequent part 2, which involves the generation of expert-based recommendations for the surveillance of SMA gene addition therapy.

Involved external institutions

Universität Duisburg-Essen (UDE) Germany (DE) Klinikum Kassel GmbH Germany (DE) Klinikum der Universität München (LMU Klinikum) Germany (DE) Kepler Universitätsklinikum (KUK) Austria (AT) Universität des Saarlandes (UdS) Germany (DE) Universitätsklinikum Bonn Germany (DE) Ruprecht-Karls-Universität Heidelberg Germany (DE) Universitätsklinikum Gießen und Marburg (UKGM) Germany (DE) Universitätsklinikum Carl Gustav Carus Dresden Germany (DE) Medizinische Hochschule Hannover (MHH) / Hannover Medical School Germany (DE) Ostschweizer Kinderspital Switzerland (CH) Universitätsklinikum Tübingen Germany (DE) Universitätsklinikum Jena Germany (DE) Charité - Universitätsmedizin Berlin Germany (DE) Medizinische Universität Innsbruck Austria (AT) Paracelsus Medizinische Privatuniversität Austria (AT) Lausanne University Hospital / Centre hospitalier universitaire vaudois (CHUV) Switzerland (CH) Universitätsklinikum Hamburg-Eppendorf (UKE) Germany (DE) Universitätsklinikum Knappschaftskrankenhaus Bochum Germany (DE) Inselspital, Universitätsspital Bern Switzerland (CH) Universitätsklinikum Freiburg Germany (DE) Universitätsklinikum Münster Germany (DE) Deutsche Gesellschaft für Muskelkranke (DGM) Germany (DE) Universitäts-Kinderspital Zürich Switzerland (CH) SMA Europe e.V. Germany (DE) Universitätsklinikum Göttingen Germany (DE)

How to cite

APA:

Weiß, C., Vill, K., Baumann, M., Bernert, G., Blaschek, A., Eisenkölbl, A.,... Ziegler, A. (2026). Delphi consensus on gene therapy of spinal muscular atrophy with onasemnogene abeparvovec in Germany, Austria and Switzerland-part I-systematic literature review and existing evidence. Journal of Neuromuscular Diseases, 13(3), 420-435. https://doi.org/10.1177/22143602251387019

MLA:

Weiß, Claudia, et al. "Delphi consensus on gene therapy of spinal muscular atrophy with onasemnogene abeparvovec in Germany, Austria and Switzerland-part I-systematic literature review and existing evidence." Journal of Neuromuscular Diseases 13.3 (2026): 420-435.

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