FDA Grants Accelerated Approval to First Drug for Rare Genetic Form of ALS

The FDA approved Biogen's drug Qalsody for some forms of ALS caused by mutations in the SOD1 gene.
The drug targets messenger RNA to reduce the toxic SOD1 protein accumulation that can lead to nerve cell death in these patients.
Qalsody will require continued study to verify its effectiveness, but shows promise for targeted treatments of ALS's genetic causes.
Out-of-pocket costs for Qalsody are expected to be under $50 per month for most patients with insurance.
Only three other drugs are approved for ALS, and Qalsody is the first drug targeting a genetic root cause of the disease.