Overview
- The Ministry of Health has added Qalsody (tofersen) to the National Health System, placing Spain among the first European countries to publicly finance the therapy.
- Indicated for ALS caused by SOD1 mutations, the treatment applies to roughly 2–3% of cases, an estimated 110 people in Spain, and is administered monthly via lumbar puncture.
- Phase III VALOR results reported functional improvement in about 25% of treated patients by week 28, alongside sustained reductions in neurofilament levels by week 148 and lower SOD1 from week 12.
- Serious intrathecal-related reactions occurred in 6.8% of participants, including myelitis, radiculitis and aseptic meningitis, though most patients continued therapy.
- Clinicians in Spain have begun treating patients with mixed early outcomes, the reported public cost is about €20,000 per patient per month, and the ATLAS study is evaluating use in presymptomatic SOD1 carriers.