Riluzole
Availability: A generic drug widely available in many countries under brand names like Rilutek, Tiglutik, and Exservan.
Approval: First approved by the U.S. Food and Drug Administration (FDA) in 1995 and has long been the only approved disease-modifying treatment in many regions, including the European Union (EU).
Mechanism: Inhibits glutamate release, a neurotransmitter that can be toxic to motor neurons at high levels.
Edaravone (Radicava)
Availability: Approved in the U.S., Canada, Japan, Switzerland, China, South Korea, and Indonesia, with both intravenous (IV) and oral formulations. The oral version, Radicava ORS, was approved in the U.S. in 2022.
Mechanism: A free radical scavenger and antioxidant that reduces oxidative stress, which is a key factor in ALS.
Tofersen (Qalsody)
Availability: An expensive, specialized therapy with limited availability for a rare, specific form of ALS.
- U.S.: Received accelerated FDA approval in 2023 for adults with a superoxide dismutase 1 (SOD1) gene mutation.
- Europe: Gained European Commission approval in May 2024 for SOD1-ALS.
Mechanism: An antisense oligonucleotide that targets the messenger RNA (mRNA) of the SOD1 gene to reduce the production of the toxic SOD1 protein.
