At Tofersen Panel Review, US FDA Shined A Light On Internal Differences Of Opinion
Agency’s presentations on Biogen/Ionis’ ALS drug included differing viewpoints from the statistical and clinical pharmacology teams; the transparency and emphasis on a multidisciplinary review approach may reflect lessons learned from missteps at the Aduhelm panel review in 2020.
You may also be interested in...
CBER Director Marks’ Intervention On Sarepta Gene Therapy Filing Decision Appears To Backfire
Former OTAT Director Wilson Bryan says Peter Marks’ order to file Sarepta’s DMD gene therapy application was mainly a public relations move, which does not seem to have worked in the FDA’s favor. Upcoming advisory committee could now involve as much scrutiny of the agency as the application itself.
Stock Watch: Full Approval And Surrogates Await Clinical Validation
When surrogate endpoints are used for accelerated approvals in advance of more compelling functional outcomes, how long should the duration of the confirmatory studies be?
Biogen Drug Sends Tau Into Reverse: An Alzheimer’s Breakthrough In The Making?
Phase I study and open-label extension results show a big drop in tau levels, which are more closely linked to Alzheimer’s symptoms than amyloid beta – setting up hopes of success in an ongoing Phase II study.