Earlier today the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee voted 6 to 4 against recommending AMX0035 for approval to treat people with ALS. It’s important to note that the Advisory Committee’s views are not binding on the FDA. Following the vote, the ALS Association called on the FDA to take into account the strong safety profile of AMX0035, as well as the serious unmet medical need of people living with this devastating condition, and approve the drug for clinical use.
Biogen and Ionis Pharmaceuticals have decided to discontinue research testing the investigational drug BIIB078 after a phase 1 clinical trial did not meet any secondary endpoints on efficacy and did not demonstrate clinical benefit.
“The Lawrence and Isabel Barnett Drug Development Program has fostered critical relationships between academia and industry and helped move the science forward. We are proud of the fact that projects supported by this program have leveraged that support to attract 6 more dollars for every $1 we initially invested,” said Dr. Kuldip Dave, vice president of research at The ALS Association.
The ALS community has the opportunity to encourage The Food and Drug Administration (FDA) to approve Amylyx Pharmaceutical’s new drug application for AMX0035.
The ALS Association has awarded $3.6 million to four interventional trials through its new Clinical Trial Awards program. The Clinical Trial Awards program is open to industry and academic investigators proposing novel or repositioning approaches for ALS.
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.
Leadership Team Member & Professor at the Medical College of Wisconsin, Dr. Ebert breaks down the challenges of finding effective therapies for ALS and how you can support the important work happening in her lab.
Research funded by The ALS Association has found that NFL players are four times more likely to be diagnosed with ALS and die from the disease than people who never played in the league, adding to the mounting evidence of a link between playing football and ALS.
We talked with Dr. Janani Parameswaran, postdoctoral fellow from Dr. Jie Jiang’s lab at Emory University in Atlanta, to learn more about her and her ALS research focused on unraveling the underlying disease mechanism.
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.