‘Qalsody (tofersen),’ a new drug for Lou Gehrig's disease, was designated as an orphan drug in Korea.

The Ministry of Food and Drug Safety (MFDS) recently announced the designation through an orphan drug designation notice.

More specifically, the drug is indicated for amyotrophic lateral sclerosis (Lou Gehrig's disease) associated with a mutation in the SOD1 (Superoxide Dismutase 1) gene.

Qalsody, which was developed by Biogen, is an antisense oligonucleotide (ASO) drug that blocks the messenger ribonucleic acid (mRNA) associated with the SOD1 gene mutation to prevent its expression.

The drug was approved by the U.S. FDA in June last year, followed by the