The FDA has approved Qalsody, an antisense oligonucleotide, to treat individuals with ALS associated with a mutation in the superoxide dismutase 1 gene.
According to an FDA press release, Qalsody (tofersen, Biogen) targets superoxide dismutase 1 (SOD1) mRNA to reduce the synthesis of SOD1 protein. The accelerated approval was based on a reduction in plasma neurofilament light (NfL), a blood-based biomarker that indicates nerve injury and neurodegeneration.
Tofersen is administered intrathecally, the FDA stated, with a recommended dose of 100 mg (15 mL) per administration. Patients receive three