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ALS Association
ALS Responds to NIH Request for Input: Speed Matters in ALS Research
The National Institute of Neurological Disorders and Stroke (NINDS) issued a request for input recently to help guide its work supporting ALS research. The ALS Association is submitting input telling the agency that speed matters. We are encouraging NINDS and the rest of NIH to focus to find ways to use research to advance the health of people with ALS as quickly as possible.
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ALS Association
ICER Set to Review ALS Treatments Like AMX0035: Here’s What We Know
The Institute of Clinical and Economic Review, commonly known as ICER, has opened a review of AMX0035 to determine the cost-effectiveness of the drug. The ALS Association is committed to making sure ICER’s review does not discriminate against people with ALS and that its analysis does not prevent people with ALS from accessing promising treatments.
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ALS Association
Dear FDA: Please Grant Priority Review to AMX0035 and Approve It
We asked the FDA to treat the approval review process of AMX0035 with urgency. Specifically, we sent a letter to FDA asking the agency to conduct a Priority Review of Amylyx’s New Drug Application (NDA) for AMX0035 and then approve it. The Priority Review is an expedited review process, as opposed to the Standard Review process, which can take upwards of a year after the agency accepts submission of the NDA.
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ALS Association
Amylyx Files NDA for AMX0035
Amylyx recently filed a New Drug Application for AMX0035, a promising new drug that has proven safe and effective at slowing progression of ALS and extending the life of people living with the disease. The ALS Association has called on the FDA to approve the application with urgency.
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ALS Association
Q&A: How Amylyx NDA Announcement Moves the Fight for Access to AMX0035 Forward
After Amylyx’s announced that it intends to file a new drug application for AMX0035, The ALS Association immediately called on the U.S. Food and Drug Administration to approve the treatment for all people with ALS as soon as possible. Connecting ALS talked to the team at Amylyx to learn about the path ahead for access to AMX0035.
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ALS Association
Case of AMX0035 Proves Advocacy from ALS Community Can Impact Drug Development
There is a lot to do, and this grounded focus of making ALS livable helps us hold everyone—ourselves, the FDA, and the research community—accountable to real impacts on real people with ALS and the time it takes to deliver those impacts. This week has been a big step forward for the ALS community, and we will continue urgently working to keep the momentum going.
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ALS Association
Our Response to the FDA’s Latest Letter and What We’re Doing to Keep Up the Pressure
Following our We Can’t Wait Action meeting with the FDA, we sent a letter to the agency asking it to reaffirm its commitment to the 2019 ALS Guidance and provide us with a detailed report showing how it has implemented the guidance. This week, the ALS Association received a response from Dr. Patrizia Cavazzoni, Director of the Center for Drug Evaluation and Research.
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ALS Association
ICYMI: We Can’t Wait Action Meeting Pushes FDA on Access
People with ALS on Tuesday called on the FDA to use its existing authority and the flexibility it promised the ALS community it would use to make experimental drugs that show incremental benefits available as quickly as possible. The comments came in a “We Can’t Wait” Action Meeting with FDA organized by The ALS Association.
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ALS Association
We Can't Wait Action Meeting with FDA: What We Heard and What's to Follow
We are  deeply grateful to the eight speakers who shared their wisdom and personal experience with the FDA,  as well as the  more than 150 others from the community  who have done so  online. We continue to hear that people with ALS want effective treatments now, even if they offer modest benefit, and are willing to face considerable uncertainty and risk to do so.
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ALS Association
ALS Association to Host FDA, Industry in “We Can’t Wait” Action Meeting
Later this month, the ALS Association will host a virtual session focused on the urgent need for timely access to new therapies. Leaders from the U.S. Food and Drug Administration (FDA) and industry companies will listen to people living with ALS speak directly about their expectations for their experiences with the disease and how important it is to have timely access to therapies that have the potential to provide incremental benefit in improving how people feel, function and live.
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ALS Association
AMX0035 Petition Delivered to FDA
The ALS Association and I AM ALS on Friday submitted a petition to the Food and Drug Administration calling on the agency and Amylyx Pharmaceuticals to act swiftly and with urgency to make AMX0035 available as soon as possible. The petition was signed by more than 50,000 people from across the country who have been affected by ALS.  
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ALS Association
A Deeper Look into ALS Association Efforts to Speed Approval of Gene Therapies
We are getting enthusiastic feedback and questions about our partnership with Project ALS to fund a clinical research project for jacifusen, an experimental therapy being developed at Columbia University’s Eleanor and Lou Gehrig ALS Center for FUS-associated ALS. We thought it would be helpful to describe the strategy underlying the project.
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ALS Association
How ALS Association Advocates Boosted Congressional Funding for ALS Research
Last week Congress passed, and the president signed, a continuing resolution that will fund all federal programs at current levels through November 21. Before the continuing resolution expires, Congress must either pass full appropriations bills for the 2020 fiscal year or pass another continuing resolution.

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ALS Association
Principles for Urgent, Patient-Centered ALS Clinical Trials Latest in Ongoing Effort to Improve Trial Design
The ALS Association is deeply committed to accelerating the development of novel ALS therapies and ensuring they are accessible to those who need them. The Association is announcing today its latest strategic action with the release of Principles for Urgent, Patient-Centered ALS Clinical Trials: a series of recommendations to clinical trial sponsors and regulatory bodies worldwide.
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