U.S. Food and Drug Administration officials said Monday that Biogen’s investigational drug ALS has shown “clinical benefit” to rare and aggressive diseases despite failing in a broader late-stage clinical trial last year. said that it could lead to Staff are at a meeting Wednesday to discuss whether a panel of external advisers should recommend early approval of Biogen’s drug Tofersen to treat a rare genetic form of amyotrophic lateral sclerosis (ALS). The FDA’s accelerated approval status is intended to allow more rapid approval of drugs for serious conditions that address unmet medical needs. The panel will review the diverse body of evidence from studies related to Tofersen’s efficacy and safety and vote on whether the drug shows clinical benefit in patients with rare ALS. FDA generally follows the advice of advisory committees, but is under no obligation to do so. “This is a situation where there is one negative clinical study that failed to demonstrate a statistically significant treatment effect in the pre-specified primary analysis population,” reads a 68-page staff report.However, Note that this study was limited in its ability to determine whether this drug would affect the overall population because of the way it was designed. This reduces “biomarkers that have been shown to correlate with disease progression and prognosis in patients with ALS.” Another exploratory analysis of the drug suggests “clinical benefit with longer duration of treatment,” co-investigators wrote. If approved, Biogen’s drug will be the first to target the genetic cause of his ALS, commonly known as Lou Gehrig’s disease, which commonly affects people between the ages of 40 and 70. It could be a product. It is a progressive, fatal neuromuscular disease that destroys nerve cells in the spinal cord and brain over time. It causes muscle spasms and spasms, severely impairs motor skills, makes swallowing and breathing difficult, and ultimately leads to paralysis and death.
