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Second Time’s The Charm: Amylyx’s ALS Drug Wins US FDA Panel Nod

 
• By Sue Sutter

In switching their votes from the first panel review in March, advisory committee members cited supportive additional analyses of a survival benefit, framing of the issues in the context of unmet need and regulatory flexibility, and the company’s statement that it would withdraw AMX0035 if the Phase III PHOENIX trial does not succeed.  

Yes and no
Amylyx flipped several votes from "no" to "yes" in its second visit to the advisory committee. • Source: Shutterstock

Amylyx Pharmaceuticals, Inc.’s amyotrophic lateral sclerosis drug AMX0035 (sodium phenylbutyrate/taurursodiol) appears on track for US Food and Drug Administration approval following a strongly favorable vote at its second advisory committee review.

In voting 7-2 for approval, four members of the Peripheral and Central Nervous System Drugs Advisory Committee switched their votes from a negative

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Maybe In May: US FDA Calls Coming On Moderna Next-Gen COVID-19 Vaccine, AbbVie’s Big ADC Bet

 
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AMR-Targeting Drug Secures EMA PRIME Designation

 
• By Neena Brizmohun

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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.

More from Regulatory Trackers

AMR-Targeting Drug Secures EMA PRIME Designation

 
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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.

Global Pharma Guidance Tracker - March 2025

 
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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.

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.