Immense research costs for a small user base. There is likely no other way to explain therapy costs of over 2 million euros including VAT per person.
Zolgensma (international designation: onasemnogene abeparvovec; ATC code M09AX09) is a gene therapy medication that has been available in Germany since 2020. SMA (spinal muscular atrophy) is a disease caused by a genetic defect and characterized by severe muscle wasting. Due to a defect in the SMN1 gene (SMN = "survival of motor neuron"), treatment with this gene therapy often remains the only viable option. In young children, administration of the medication can delay the progression of the disease. The initial price set by the manufacturer, Novartis/AveXis, is widely considered to be significantly excessive.
Administration typically takes place as a single intravenous infusion, with a total treatment duration of approximately 60 minutes. The treatment must be performed in specialized clinical centers by experienced physicians. Prior assessment of liver function values and antibody levels is required. Acute liver failure may occur as a side effect, rendering this drug therapy potentially hazardous. However, Zolgensma can halt the progression of muscle weakness and facilitate improvements in motor function. In no case does Zolgensma eliminate the need for continued physiotherapy.
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Sources
Magazin Welt der Wunder im Artikel: "Was ist das teuerste Medikament der Welt", Ausgabe 12/24, S. 30 (German)
Onlineportal der Betriebskrankenkassen (BKK e.V.) im Artikel: "Das teuerste Arzneimittel der Welt ist nun in Deutschland auf dem Markt", abgerufen Dez, 2024 (German)
Dr. Frank Antwerpes, Michael Vogt im Artikel für doccheck-com: "Onasemnogen-Abeparvovec", abgerufen Dez, 2024 (German)
flexikon.doccheck.com/de/Onasemnogen-Abeparvovec