The Ministry of Food and Drug Safety announced on the 20th that it has approved the domestic use of the drug 'Qalsody' (generic name: Tofersen) for the treatment of amyotrophic lateral sclerosis (ALS, Lou Gehrig's disease).
Qalsody is a new drug for Lou Gehrig's disease developed by Biogen in the United States, which received approval from the U.S. Food and Drug Administration (FDA) in 2023. It was subsequently approved by Europe in May last year.
Lou Gehrig's disease is a fatal condition characterized by the gradual loss of motor neurons, leading to symptoms such as muscle weakness, atrophy, speech difficulties, limb weakness, weight loss, and pneumonia, ultimately resulting in respiratory failure and death. It is named after the famous American baseball player Lou Gehrig, who suffered from this disease in 1939.
Qalsody is the first treatment to be administered to patients with SOD1 gene mutations. It blocks the mutated SOD1 gene using ribonucleic acid (RNA).
The Ministry of Food and Drug Safety designated Qalsody, a treatment for rare diseases, as the 31st product under the Global Innovative Product Fast-Track Review System (GIFT), granting approval through an expedited review process. A Ministry official noted, "We will continue to do our best to ensure that treatments with confirmed safety and efficacy are reviewed and approved quickly, expanding treatment opportunities for patients."