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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.

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More from US FDA Performance Tracker

AMR-Targeting Drug Secures EMA PRIME Designation

 
• By Neena Brizmohun

Antabio’s next-generation, broad-spectrum antibacterial, MEM-ANT3310, has made it onto the European Medicines Agency’s priority medicines scheme. The company says its product is designed to make a significant impact on the growing problem of antimicrobial resistance.

US FDA’s Review Performance Held Steady Despite Stormy First Quarter

 
• By Bridget Silverman

Organizational turmoil has not yet affected reviews of novel agent applications, with Q1 2025 approvals coming in low, but the first quarter share of the entire year is variable.

‘Pipeline In A Pill’ Or Pipe Dream? US FDA’s April Goal Dates Test Expansion Strategies

 
• By Bridget Silverman

Sanofi’s Dupixent, Amgen’s Uplizna, and Bristol’s Opdivo seek new indications, while J&J hopes to start a franchise with nipocalimab and Stealth’s day of reckoning approaches.

US FDA’s March Approval Candidates Take Aim At Established Markets

 
• By Bridget Silverman

March features nearly 20 user fee goal dates, including a crowd of novel candidates for crowded areas like hemophilia, heart disease and urinary tract infections, as well as the potentially first therapies for a rare eye disease and hyperphagia of Prader-Willi syndrome.

More from Regulatory Trackers

‘Pipeline In A Pill’ Or Pipe Dream? US FDA’s April Goal Dates Test Expansion Strategies

 
• By Bridget Silverman

Sanofi’s Dupixent, Amgen’s Uplizna, and Bristol’s Opdivo seek new indications, while J&J hopes to start a franchise with nipocalimab and Stealth’s day of reckoning approaches.

Global Pharma Guidance Tracker – February 2025

 
• By Vibha Sharma and Anabel Costa-Ferreira

Stay up to date on regulatory guidelines from around the world with the Pink Sheet's Guidance Tracker. The complete Global Pharma Guidance Tracker, with sortable and searchable listings going back to 2014, is available online.

EU CHMP Opinions And MAA Updates

 
• By Neena Brizmohun

This is an update of recommendations from the European Medicines Agency's Committee for Medicinal Products for Human Use on the authorization of new medicines in the EU, and updates on EU marketing authorization changes recommended by the CHMP.