Latest From Sue Sutter
Accelerated Approval: Potential Sources Of Confirmatory Evidence Weighed At Tofersen Review
‘Clear signal’ in the ATLAS study of presymptomatic, SOD1 mutation carriers could provide evidence needed to confirm tofersen’s benefit in symptomatic ALS patients, FDA’s Teresa Buracchio says, adding that getting confirmatory evidence from the VALOR open-label extension 'could be a little more tricky.'
Accelerated Approval Is US FDA Panel’s Preferred Path For Biogen/Ionis’s Tofersen In ALS
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.
Electronic Records: US FDA Updates Guidelines On Use In Clinical Investigations
Revised draft guidance includes recommendations on preparing for FDA inspections, considerations for real-world data and digital health technologies, and the need for service level agreements between sponsors and IT providers.
Biogen/Ionis’ Tofersen: US FDA Considering Both Accelerated And Regular Approval In ALS
FDA seeks advisory committee input on whether there is ‘convincing evidence’ to support regular approval for treatment of SOD1-ALS patients even though lone Phase III trial failed its primary endpoint; companies filed for accelerated approval on the basis that a reduction in plasma neurofilament light chain is reasonably likely to predict clinical benefit.
Sarepta’s DMD Gene Therapy Adcomm Likely To Focus On Dystrophin As A Surrogate Endpoint
Cellular, Tissue and Gene Therapies Advisory Committee will weigh whether the increase in dystrophin production seen with SRP-9001 is reasonably likely to predict clinical benefit in Duchenne muscular dystrophy, Sarepta says; agency had previously told the company a panel meeting would not be needed, but it reversed course late in the review.
Genentech’s Polivy Gets US FDA Panel Nod For First-Line Use In Large B-Cell Lymphoma
Advisory committee members were swayed by a progression-free survival benefit, and the reduction in salvage therapies, with a polatuzumab-containing regimen relative to the highly effective standard of care in the POLARIX trial.