Ministry of Food and Drug Safety Approves ALS Treatment "Qalsodyju"

Expanding Treatment Opportunities for Rare Diseases

The Ministry of Food and Drug Safety announced on August 20, 2025, that it has approved the import of the rare disease treatment "Qalsodyju (Tofersen)."

This drug is a nucleic acid therapy that binds to SOD1 mRNA in patients with amyotrophic lateral sclerosis (ALS) who have a superoxide dismutase 1 (SOD1) gene mutation, thereby reducing the synthesis of the mutated superoxide dismutase 1 (SOD1).

SOD1 is a protein with antioxidant properties that acts as an enzyme to protect nerve cells from damage caused by reactive oxygen species, but mutations can lead to neurological diseases. Amyotrophic lateral sclerosis is a severe and rare disease that causes muscle weakness and gradually leads to motor dysfunction.

The Ministry of Food and Drug Safety expressed hope that the approval of Qalsodyju will provide a new treatment option for adult patients with SOD1 gene-mutated amyotrophic lateral sclerosis who currently lack appropriate therapies. An official from the Ministry stated, "After designating this treatment as the 31st product under the Global Innovative Product Fast Track (GIFT) system, we conducted an expedited review to ensure its prompt introduction into the domestic healthcare field."

This content was produced with the assistance of AI translation services.