FDA Approves First ALS Treatment Funded by Ice Bucket Challenge

The Golden West Chapter celebrates the Food and Drug Administration’s (FDA) announcement of approval of RELYVRIO (formerly AMX0035) as a new treatment for adults living with amyotrophic lateral sclerosis (ALS), a fatal neurodegenerative disease.

The ALS Association invested $2.2 million of funds, raised through the 2014 ALS Ice Bucket Challenge into the development and trial of AMX0035, and led the years-long advocacy campaign that pushed the FDA to approve the treatment, prior to completion of an ongoing phase 3 trial. 

“The approval of RELYVRIO is further proof of how the Ice Bucket Challenge dramatically accelerated the fight against ALS,” said Sheri Strahl, Chief Operating Officer of the Golden West Chapter. “Since this unprecedented, grassroots outpouring of support, we’ve seen new genes discovered, new assistive technology developed to help people living with ALS, and far more people living with ALS and their families have access to care services than ever before.”

The company announced the news with great appreciation to the many individuals and organizations who supported their efforts,

“Today’s FDA approval of RELYVRIO is an exciting milestone for the ALS community and is a major step toward achieving our mission to one day end the suffering caused by neurodegenerative diseases,” said Joshua Cohen and Justin Klee, Co-CEOs of Amylyx.

“We want to give a heartfelt thank you to the broader ALS community, including healthcare professionals and those living with ALS, for their guidance, support of our clinical programs, and for sharing their experiences with us.“

“Their stories inspired us and helped our team to better understand the ALS clock, instilling in us a deep sense of urgency that will continue to drive us forward. This is just the beginning, and there is much more to be done.”

In the summer of 2014, over 17 million people posted Ice Bucket Challenge videos to Facebook, including hundreds of celebrities, athletes, and politicians. The campaign, which was launched by Anthony Senerchia Jr., Pat Quinn, and Pete Frates – three young men who were living with ALS – raised over $115 million for The ALS Association. 

Since the Ice Bucket Challenge, The ALS Association has invested over $127 million in ALS research and is currently funding 130 research projects in 12 countries. The Association is currently funding 40 potential treatments in the drug development pipeline. In addition, the Association has expanded its network of clinical providers from 100 Certified Treatment Centers of Excellence, Recognized Treatment Centers, and Affiliated Clinics in 2014 to 188 today.

It was in June of 2016 that The ALS Association provided Amylyx, the producer of AMX0035, 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.

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 Action Meeting in May of 2021, so that members of the ALS community could speak directly to FDA officials. The Association also provided scientific and regulatory guidance, and in 2022, drove over 14,000 individual emails to the FDA urging approval. 

The Golden West Chapter also worked closely with Amylyx and many of our families to provide direct feedback and personal stories that ultimately helped contribute to this outcome. Thousands of people living with ALS, their loved ones, and advocates sent emails to the FDA urging the agency to approve the treatment. The Chapter mobilized ALS advocates who participated in several meetings with FDA officials.

The Chapter recognized Amylyx with our 2021 Commitment to Cures Award for their efforts to champion drug discovery and development in the relentless pursuit of treatments and cures for people with ALS and related diseases.

The Golden West Chapter is working closely with Amylyx and our certified ALS clinics and centers to monitor and advocate for access and availability of the drug to our ALS community. Please visit our FAQ’s about RELYVRIO webpage for more information.