US FDA’s ALS Science Strategy Includes Near-Term Rare Neurodegenerative Disease Task Force
Five-year plan includes multi-phased approach to ALS at the same time that Amylyx’s AMX0035 is pending review with a late September decision deadline; action plan’s mid-term priorities for FY 2023-2024 include a cell and gene therapies safety project.
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ALS Drugs: FDA May Adjust Development Recommendations In Real Time For Ongoing Programs
Agency’s action plan for rare neurodegenerative diseases maps out over five years, but knowledge gained can inform products currently in development; new FDA grant program looks to invest in remote use of clinical outcome assessments for amyotrophic lateral sclerosis.
ALS ‘Urgency’, US FDA Regulatory Environment Justify Filing NurOwn, BrainStorm Says
BrainStorm discovered a statistical error while preparing the stem cell therapy's BLA, and corrected data show a statistically significant treatment difference for a key secondary endpoint in less advanced ALS. However, the Phase III trial still failed its primary clinical efficacy endpoint.
Here We Go Again: US FDA Weighing Accelerated Approval For Biogen’s ALS Drug Tofersen
Company seeks accelerated approval based on changes in neurofilament as a surrogate for efficacy; with controversy over FDA’s Aduhelm approval in Alzheimer’s still raging, agency is again faced with deciding whether biomarker data in a neurodegenerative disease with high unmet need are sufficient to justify approval in the face of a failed clinical endpoint study.