TOFERSEN (Qalsody®) is an antisense oligonucleotide that binds the so-called messenger RNA of SOD1 (superoxide dismutase 1) and thus significantly reduces the expression of the SOD1 protein. In a pivotal study on SOD1 patients with different mutations in the SOD1 gene, not only was there a reduction in the axonal damage maker neurofilament, but also a slowing of motor decline, measured on the ALS-FRS scale, as well as a prolongation of survival. The drug had therefore been approved by the FDA for the treatment of SOD1-ALS since 2023. Previously, SOD1 patients in Germany had the opportunity to participate in an open access programme run by Biogen. Around 40 patients across Germany have already been successfully treated in this programme. In May 2024, the EMA has now also granted marketing authorisation for Europe. This means that the open access programme will end in June 2024 and the drug will be available in the classic form from 1 July 2024.

Similar to ASO therapy for spinal muscular atrophy (Spinraza®), the drug must be injected into the spinal canal (intrathecal administration). This is done 3 times in the first month, then at 28-day intervals. Experience to date in the German network shows that the treatment is well tolerated by most patients.