Biogen Inc.’s studies of its drug for a rare form of amyotrophic lateral sclerosis suggest the drug has a “treatment effect,” the Food and Drug Administration’s staff said in documents released ahead of a meeting out of outside advisers this week.
(Bloomberg) — Biogen Inc.’s studies of its drug for a rare form of amyotrophic lateral sclerosis suggest the drug has a “treatment effect,” the Food and Drug Administration’s staff said in documents released ahead of a meeting out of outside advisers this week.
The agency’s staff recognizes the disease is a “severely debilitating and life-threatening disease with substantial unmet need” and that patients are willing to accept risk given the severity of the condition, according to the documents released on the FDA website Monday. Although the drug, tofersen, failed the main goal of a final-stage trial, “there appear to be generally consistent trends favoring tofersen across different statistical methods,” the staff wrote.
The FDA is also weighing what level of approval would be supported by the company’s data that show the drug decreased levels of a protein associated with brain injury. The independent panel of advisers to the FDA will review the data and vote on a recommendation in a meeting Wednesday.
Shares of Biogen rose 0.9% Monday at 10:07 a.m. in New York.
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