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.
Accelerating Access to Critical Therapies (ACT) for ALS (H.R. 3537) was approved by a unanimous vote on Thursday, November 4 in the House Energy and Commerce Subcommittee on Health. Thanks to everyone within the ALS community who has advocated for passage and who has worked on Capitol Hill to help make this important step happen. The strong bipartisan support for this bill means smooth sailing as the bill goes to the full House Energy and Commerce Committee and then the full House for approval.
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.
Unlike most Medicare recipients who need extensive home care and rehabilitative services, people with ALS do not improve, and the intensity of their service needs increase over time. That means standard Medicare cost control approaches don’t work well. I will discuss two examples.
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.
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.
Thanks to the tireless efforts of ALS advocates across the country, the House included many of our aggressive requests for new and increased investments in research to find treatments and a cure, slow symptom progression, reduce the number of new cases, and increase the length and quality of life for every American living with ALS.
In very powerful testimony, members of the ALS community asked members of Congress to ensure the FDA acts with greater speed and regulatory flexibility during a hearing before the House Energy and Commerce Health Subcommittee.
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.
ALS advocates are pursuing an aggressive set of public policy priorities for people living with ALS and their families. These priorities include increased federal funding for ALS research, creating new pathways for expedited approval of promising treatments including the Promising Pathway Act and ACT for ALS, making permanent expanded access to telehealth, and increasing access to high-quality health care and veteran's benefits.
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.
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.
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.
Recent changes to Medicare will enable people with ALS to receive services from speech language pathologists via telehealth through the end of the COVID-19 pandemic. These services include clinical care for swallowing and speech-generating devices - many challenges people living with ALS are faced with every day.
Recent changes at the Centers for Medicare & Medicaid Services (CMS) will enable people with ALS to receive critical services provided by speech therapists, normally provided at in-person visits, via telehealth during the pandemic. These services include clinical care for swallowing and speech-generating devices - many challenges people living with ALS are faced with every day.
“Shortly after being diagnosed, I began researching and discovered how devastating this disease really is,” Pattie says. “In those early months I struggled emotionally with the outlook that was now my reality. Eventually, I chose to use my situation as motivation to try and make a difference for the future of ALS.”
The ALS Association supports ACT for ALS and the Promising Pathway Act as they were originally introduced in the last Congress. Following the 2020 election, a new Congress was sworn-in in January of 2021. The legislative process requires that both bills be reintroduced to the newly elected Congress, and we are working with our congressional champions to reintroduce them as soon as possible. We believe both bills will make a positive impact on people living with ALS and look forward to working with the community and Congress to pass them.
On December 22, the ALS Disability Insurance Access Act became law. Since the law was passed, The ALS Association has met with the Social Security Administration to ensure applications for SSDI are quickly processed and benefits start immediately for people with ALS.
The ALS Disability Insurance Access Act went into law Tuesday, removing the 5-month delay for people with ALS to access the Social Security Disability benefits. This marks the culmination of a multi-year fight by ALS advocates across the country.