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One Difference Between Amylyx’s ALS Drug And Aduhelm: A Biomarker Endpoint

 
• By Brenda Sandburg

US FDA’s Billy Dunn says that if the drug had shown a positive effect on plasma neurofilament heavy chain, it ‘would have provided important contextual and supportive information of an ostensibly beneficial effect on the clinical measure.’

ALS
Biomarker data could have helped show efficacy of Amylyx's ALS drug AMX0035 • Source: Alamy

Amylyx Pharmaceuticals, Inc.’s drug AMX0035 for treatment of amyotrophic lateral sclerosis will not be following the approval pathway of Aduhelm, at least not based on a surrogate endpoint. US Food and Drug Administration officials noted at an advisory committee meeting that there is no established biomarker for AMX0035 but that it would have been helpful to “the contextual picture” if AMX0035 had been shown to have an impact on a promising potential biomarker.

The agency granted accelerated approval for Biogen, Inc./Eisai Co., Ltd.'s Aduhelm (aducanumab) based on a single positive Phase III study. The agency concluded there was substantial evidence that the drug reduces amyloid beta plaque in the brain and that this surrogate endpoint is reasonably likely to predict clinical benefit

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Keep Talking: US FDA’s Beleaguered Biologics Center Remains Vital To Regenerative Medicine

 
• By Bridget Silverman

Advisory committee for Capricor’s deriamocel planned, regenerative medicine advanced therapy designations proliferate for osteoarthritis, and the ranks of dual RMAT/breakthrough therapy designation holders grow.

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

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.

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

 
• 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.

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.