The U.S. Food and Drug Administration has approved a new drug to treat a rare form of Amyotrophic Lateral Sclerosis (ALS), also commonly known as Lou Gehrig’s disease.
The injectable drug can now be used for patients with a very specific mutation which affects fewer than 500 people with ALS in the United States.
The drug, Qalsody (tofersen), is the first of its kind intended to treat an inherited type of ALS with mutations in the SOD1 gene that cause the breakdown of nerve cells essential for basic functions.
According to the Centers for Disease Control and Prevention, between 17,000 to nearly 32,000 Americans currently living with ALS, but only 2% of ALS cases are associated with mutations in the SOD1 gene.
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The approval was granted through the FDA's accelerated pathway, which enables drugs to be launched based on promising initial findings prior to being fully confirmed to have benefits for patients.
"The approval was based on a reduction in plasma neurofilament light (NfL), a blood-based biomarker of axonal (nerve) injury and neurodegeneration," the FDA said in a press release. "The findings are reasonably likely to predict a clinical benefit in patients. The observed reduction in NfL was consistent across all subgroups based on sex, disease duration since symptom onset, site of onset, and use of other medications for ALS treatment."
However, the FDA wants the drug's maker, Biogen, to keep studying the drug in a trial of people carrying the genetic mutation who have not yet displayed symptoms of ALS.
The drug is administered via a spinal injection in three doses at 14-day intervals, followed by a dose every 28 days, according to the FDA.
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