Accelerated Approval Is US FDA Panel’s Preferred Path For Biogen/Ionis’s Tofersen In ALS
Executive Summary
Advisory committee unanimously concludes that the reduction in plasma neurofilament light chain concentration is reasonably likely to predict clinical benefit in SOD1-ALS, but majority of panelists say convincing evidence of efficacy to support regular approval has not been demonstrated.
You may also be interested in...
US FDA Neuroscience Office In Spotlight With Donanemab Delay, Relyvrio Trial Failure
Lilly’s surprise announcement that the Alzheimer’s drug is going to an advisory committee, followed minutes later by Amylyx’s disclosure that its ALS drug failed a Phase III study, marked a one-two punch of bad news for the Office of Neurosciences’ recent use of regulatory flexibility.
AstraZeneca’s Danicopan Among Four Orphans Up For EU Approval Decision
The European Medicines Agency is due to decide whether to back pan-EU marketing authorization for 11 new products, including four orphan drugs.
Cell/Gene Therapies: US FDA Wants ‘Quantitative Correlation' Between Surrogate And Clinical Outcome, Former Reviewer Says
When weighing accelerated approval, the FDA wants to see that ‘X change in the surrogate translates into Y change in the clinically meaningful outcome,’ Parexel’s Steve Winitsky says. CBER Director Peter Marks opines on the types of therapeutic settings where accelerated approval is best suited, and what happens when a confirmatory trial disappoints.