The U.S. Food and Drug Administration on Tuesday authorized a drug for a rare genetic form of the neurological disorder amyotrophic lateral sclerosis, commonly called Lou Gehrig’s disease.
The drug, which is known as tofersen, will be sold under the brand name Qalsody. The drug targets a mutation in a gene known as SOD1.
According to the Centers for Disease Control and Prevention, between 16,000 and 32,000 Americans are currently living with ALS with approximately 2% of those ALS cases associated with mutations in the SOD1 gene, the FDA said in a statement.
Fewer than 500 people in the United States at any given time are expected to be eligible for the drug.
According to The New York Times, Biogen, the pharmaceutical company bringing the drug to market, said it would price the drug “within a range comparable to other recently launched ALS treatments.”
An ALS therapy approved last year was priced at $158,000 annually, according to the Times.
The FDA granted accelerated approval of the drug via a policy that allows a drug to be fast-tracked onto the market if it meets certain criteria and if there is conclusive proof that it works. Under the approval classification, Biogen will be required to continue clinical research into Qalsody’s effectiveness in order to keep it on the market.
In a clinical trial, the drug did not significantly slow the progression of the disease, as measured by a patient’s ability to speak, swallow, walk and perform other activities of daily living, according to the Times.
Biogen said it believes that the drug will show in future trials that it is effective in slowing the progression of damage from the disease for those with that specific type of ALS.
