Pink Sheet is part of Pharma Intelligence UK Limited

This site is operated by Pharma Intelligence UK Limited, a company registered in England and Wales with company number 13787459 whose registered office is 5 Howick Place, London SW1P 1WG. The Pharma Intelligence group is owned by Caerus Topco S.à r.l. and all copyright resides with the group.

This copy is for your personal, non-commercial use. For high-quality copies or electronic reprints for distribution to colleagues or customers, please call +44 (0) 20 3377 3183

Printed By

UsernamePublicRestriction

Ionis Pharmaceuticals, Inc.

http://www.ionispharma.com

Latest From Ionis Pharmaceuticals, Inc.

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.'

Advisory Committees Review Pathway

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.

Advisory Committees Drug Review

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.

Advisory Committees Drug Review

Scrip Asks…What Does 2023 Hold For Biopharma? Part 8: Therapeutic Area Advances

Neuroscience and oncology fuel high hopes for therapeutic R&D progress among 90-plus surveyed biopharma industry leaders.

Scrip Asks Research & Development
See All

Company Information

  • Industry
  • Biotechnology
  • Pharmaceuticals
  • Pharmaceuticals
    • Drug Delivery
  • Biotechnology
    • Antisense, Oligonucleotides
    • Drug Discovery Tools
    • Large Molecule
  • Other Names / Subsidiaries
    • Akcea Therapeutics, Inc.
    • Regulus Therapeutics, Inc.
    • Symphony GenIsis, Inc.
    • Isis Pharmaceuticals, Inc.
UsernamePublicRestriction

Register