The FDA has long used accelerated approval to speed the availability of drugs for cancer and other deadly conditions.
The FDA is requiring Biogen to continue studying the drug in a trial of people who carry the genetic mutation but do not yet have ALS symptoms.
The drug, tofersen, is designed to block the genetic messengers that produce a toxic form of protein that is thought to drive the disease in about 2% of ALS patients. Cambridge, Massachusetts-based Biogen will sell it under the brand name Qalsody. Patients receive three initial spinal injections of the drug over a two-week period, followed by a monthly dose. The most common side effects linked to the drug were pain, fatigue and increased spinal fluid.
But those who received tofersen showed significant changes in levels of the toxic protein and a second neurological chemical that is considered a key indicator of the disease's progression.
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