1. Pink Sheet
  2. >>Regulatory Trackers

Accelerated Approval Is US FDA Panel’s Preferred Path For Biogen/Ionis’s Tofersen In ALS

 
• By Sue Sutter

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.

Edge of cliff
Most FDA adcomm members were unwilling to make the leap to regular approval for tofersen. • Source: Shutterstock

The available evidence supports accelerated approval, but not regular approval, for Biogen, Inc. and Ionis Pharmaceuticals, Inc.’s Qalsody (tofersen) in a subgroup of patients with amyotrophic lateral sclerosis, a US Food and Drug Administration advisory committee said on 22 March.

The Peripheral and Central Nervous System Drugs Advisory Committee voted unanimously that a reduction in plasma neurofilament light chain (NfL) concentration with tofersen is reasonably likely to predict clinical benefit...

Read the full article – start your free trial today!

Join thousands of industry professionals who rely on Pink Sheet for daily insights

  • Start your 7-day free trial
  • Explore trusted news, analysis, and insights
  • Access comprehensive global coverage
  • Enjoy instant access – no credit card required
Start Free Trial

Already a subscriber?

More from US FDA Performance Tracker

More from Regulatory Trackers