ALS Association Applauds Amylyx AMX0035 Announcement, Urges Swift FDA Approval

Association was early funder of potential new ALS drug, advocated for early approval

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Sep 15, 2021, 08:15 ET

WASHINGTON, Sept. 15, 2021 /PRNewswire/ -- Following Amylyx's announcement that it intends to submit a New Drug Application (NDA) for AMX0035, The ALS Association today urges the Food and Drug Administration to approve the treatment for all people with ALS as soon as possible. The Association was an original funder of Amylyx and has advocated for early approval of AMX0035, since the results of a trial showed the treatment slowed progression in people with ALS and an open-label extension study showed an increase in life among those on the drug.

AMX0035, a promising new treatment for ALS, soon to be submitted for FDA approval, is an outcome of funding received through the ALS Ice Bucket Challenge. “We continue to see the impact of the millions of people around the world who took the Challenge in the summer of 2014 and we are forever grateful to the people with ALS and families who started it. They truly changed the trajectory of ALS forever.” --ALS Association President & CEO Calaneet Balas.

"We are excited that people with ALS may soon have another treatment option that will help make ALS livable," said Calaneet Balas, president and CEO of The ALS Association. "We are further encouraged that the FDA appears to have finally heard the loud and clear message from the ALS community that people with ALS are willing to accept greater risk to try potential treatments. We thank everyone who advocated and shared their voice with the FDA and urge the agency to approve AMX0035 swiftly."

In September of 2020, The ALS Association and I AM ALS submitted over 50,000 signatures to the FDA calling on the agency to approve AMX0035. In subsequent months, the Association held multiple meetings with FDA officials, including a public We Can't Wait Action Meeting in May of 2021, so members of the ALS community could speak directly to FDA officials.

"The ALS community has been united in this effort and we expect the FDA to act quickly," said Larry Falivena, who is living with ALS and is a member of the Association's Board of Trustees. "Given the promise that AMX0035 shows in slowing progression and its safety record, the community should not have to wait several more years for another clinical trial. Thousands of us who could have been helped will have passed from this devastating disease."

The ALS Association's funding for AMX0035 was supported through donations received through the ALS Ice Bucket Challenge. In June of 2016, the Association provided Amylyx with a $750,000 grant for a clinical trial pilot, and in July of 2016, the Association provided the Northeast ALS Consortium (NEALS) with a $1.46 million grant to help pay for the phase 2 clinical trial of AMX0035. The Association has also provided regulatory, medical, and research counsel to help expedite the progress of AMX0035.

"The Ice Bucket Challenge led to new ALS genes being discovered, greater access to care services for people with ALS and their families, and in AMX0035, a very promising new treatment that could make a difference for thousands of people with ALS," said Balas. "We continue to see the impact of the millions of people around the world who took the Challenge in the summer of 2014 and we are forever grateful to the people with ALS and families who started it. They truly changed the trajectory of ALS forever."

Following Amylyx's submission of its NDA for AMX0035, the FDA will determine whether to grant approval of the drug or require additional study, a process that could take several months. According to the company, Amylyx has also filed for approval of AMX0035 in Canada and will seek approval in the European Union by the end of the year.

As a standard provision in philanthropic support for drug development, the grants to Amylyx and for the clinical trial of AMX0035 included repayment provisions allowing the Association to recover up to 150 percent of its support, or up to $3.3 million. Any funds received will be reinvested into ongoing global research into treatments and cures.

About ALS
Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease that afflicts approximately 16,000 people in the United States and a comparable number of patients in Europe. Approximately 5,000 new cases of ALS are diagnosed each year in the United States. The average life expectancy of an ALS patient is approximately three to five years after diagnosis and only approximately 10 percent of patients survive for more than 10 years. Death is usually due to respiratory failure because of diminished strength in the skeletal muscles responsible for breathing. Few treatment options exist for these patients, resulting in a high unmet need for new therapies to address functional deficits and disease progression.

About The ALS Association
The ALS Association is the largest private funder of ALS research in the world. The Association funds global research collaborations, provides assistance for people with ALS and their families through our nationwide network of chapters and certified clinical care centers, and advocates for better public policies for people with ALS. The ALS Association builds hope and enhances quality of life while urgently searching for new treatments and a cure. For more information about The ALS Association, visit our website at www.als.org.

SOURCE The ALS Association