(Reuters) -A panel of U.S. Food and Drug Administration (FDA) advisers on Wednesday voted against the effectiveness of Biogen Inc’s experimental treatment for an inherited form of amyotrophic lateral sclerosis (ALS).
The FDA panel voted 5 to 3 against the drug, tofersen, as an effective treatment for ALS patients with mutations in a specific gene that leads to accumulation of toxic levels of a protein known as superoxide dismutase 1 (SOD1).
One panelist abstained from voting on the effectiveness of the drug.
“I think it (the data) meets the evidentiary standards for accelerated approval but not for full approval, which is essentially what we’re being asked here,” said panelist Robert Alexander, who voted against the effectiveness of the drug.
The panel also voted on data, which showed that tofersen reduced levels of the protein neurofilament in patients. Advisers voted unanimously in favor of the protein-lowering data as sufficient evidence of the drug’s benefits for patients.
ALS, the cause of which is largely unknown, is also known as Lou Gehrig’s disease. The disease results in weakened muscles and causes nerve cells in the brain and spinal cord to break down, affecting physical function and leading to severe disability and death.
(Reporting by Bhanvi Satija and Raghav Mahobe in Bengaluru; Editing by Shounak Dasgupta)
