Urgency Meets Opportunity: ALS Community Calls for Expedited FDA Pathways as Promising Therapies Advance
A Citizen Petition and Potential for New Drug Application Offer Promise for a 100% Fatal Disease
FOR IMMEDIATE RELEASE: July 7, 2025, Washington, D.C. – With two investigational ALS treatments making critical regulatory strides, advocates are calling on the Food and Drug Administration (FDA) to honor its commitment to urgency and flexibility in reviewing therapies for this 100% fatal disease. This moment marks a turning point in the fight for access, hope, and time.
In late June, BrainStorm Cell Therapeutics’ investigational cell-based therapy NurOwn took a significant step forward after ALS advocates filed a citizen petition requesting a new FDA review. Citizen petitions carry weight, compelling a formal agency response and signaling the collective voice of a community demanding action.
BrainStorm also released encouraging new survival data from its Expanded Access Program (EAP), one of the few current avenues allowing people with ALS to receive experimental therapies. This data presents an immediate opportunity for the FDA to apply its own guidance around regulatory flexibility for serious and life-threatening diseases.
“ALS is relentless. So are we,” said Andrea Goodman, CEO of I AM ALS. “With a life expectancy of just 2–5 years after diagnosis, patients can’t wait. We need science to move faster—and we need the FDA to meet that urgency with meaningful action.”
Meanwhile, a second ALS therapy in development—CNM-Au8—continues to show promise. Clene Nanomedicine announced they recently completed one FDA meeting and have two more scheduled to discuss survival data and its MS development program. The FDA has also provided supportive feedback on Clene’s statistical analysis plan for neurofilament light (NfL) biomarker data from its NIH-sponsored EAP, with analyses beginning in Q4 2025. If results are favorable, Clene plans to submit a New Drug Application for CNM-Au8 under the accelerated approval pathway. This forward momentum underscores the critical role real-world evidence—especially from EAPs—can play in shaping regulatory decisions for terminal diseases like ALS.
“We fully support the fast review of both NurOwn and CNM-Au8, as well as other promising therapies in the pipeline. In both cases we’re seeing unprecedented survival data – this cannot be ignored in ALS. These are not just case studies in innovation—they’re roadmaps for what’s possible when science, urgency, and patient advocacy converge. This is what is going to turn hope into survival,” said Goodman.
I AM ALS continues to lead efforts to ensure faster, rigorous regulatory pathways that reflect the dire realities of those living with ALS. EAPs are more than a research mechanism—they’re a lifeline for people living with no approved options and no time to waste.
This moment demands more than optimism—it demands action. Advocates are urging the FDA to use every tool at its disposal to expedite reviews, embrace flexibility, and ensure that treatments that are working reach as many people who need them as possible.
About I AM ALS
I AM ALS is a nonprofit organization leading what STAT News called the most successful patient advocacy campaign this century. We built a community movement to harness collective power and find treatments and a cure for ALS faster, while also creating lasting, systemic change. Our focus is on three areas:
- Advocating for federal policy change to drive research, support, and treatments for ALS.
- Improving quality of life by providing volunteer and support opportunities to advocates and people living with ALS.
- Mobilizing and empowering advocates to raise awareness about ALS and other neurodegenerative diseases, and increase visibility of the ALS experience.
Learn more at www.iamals.org.