Biogen (NASDAQ: BIIB)’s Chinese subsidiary has announced that it has received conditional approval from the National Medical Products Administration (NMPA) for its drug Qalsody (tofersen) for the treatment of adult patients with amyotrophic lateral sclerosis (ALS) who have a superoxide dismutase 1 (SOD1) gene mutation. ALS is a rare disease, and the SOD1 gene was the first pathogenic gene identified for it. Patients with SOD1-ALS often experience spinal cord onset, which is characterized by simultaneous damage to both upper and lower motor neurons.
Qalsody, an antisense oligonucleotides (ASO) drug, is recognized as the world’s first disease-modifying therapy for SOD1-ALS and the first precision medicine for ALS in China. It works by reducing the synthesis of the SOD1 protein and decreasing the accumulation of toxic SOD1 protein, which can alleviate damage to motor neurons and slow down the progression of ALS. – Flcube.com
