- The Food and Drug Administration and National Institutes of Health launched a public-private initiative focused on better understanding rare brain conditions such as amyotrophic lateral sclerosis and developing new medicines for them.
- Called the Critical Path for Rare Neurodegenerative Diseases, the program is being run by the Critical Path Institute, a nonprofit created in 2005 that works closely with the FDA to improve and accelerate development of innovative medicines. The effort will include scientists, patient communities, advocacy organizations and private entities, the FDA said Wednesday.
- The initiative is part of a law signed late last year by President Joe Biden that focused on amyotrophic lateral sclerosis, or ALS. The law allocated $100 million over five years for the public-private partnership as well as grant programs run by the FDA and the U.S. Department of Health and Human Services.
There’s been little progress in fighting ALS since 1939, when New York Yankees star Lou Gehrig retired from baseball because of the disease. Patients with the condition become paralyzed, and most die within two to five years of diagnosis.
Only two main treatments are approved to specifically address the progression of the disease, though neither can reverse its effects. Rilutek, cleared by the FDA in 1995, has been shown to increase life expectancy by about three months. Mitsubishi Tanabe Pharma’s Radicava, approved in 2017, can help reduce the decline in daily functioning experienced by patients.
Between 1995 and 2017, researchers studied more than 60 molecules as potential treatments for the disease. The overwhelming majority failed.
The history of setbacks has led to dramatic scenes such as an FDA meeting in 1997, when patients and their family members wept after an agency advisory panel voted to reject an experimental drug called Myotrophin because of conflicting study data.
More recently, the FDA has signaled that it will be more flexible on ALS drugs. It convened a rare second advisory committee meeting for an Amylyx Pharmaceuticals medicine after the panel initially voted against it. This time, the FDA’s outside experts recommended approval of AMX0035 after FDA official Billy Dunn emphasized the agency’s duty to engage in “regulatory flexibility.”
In July, Biogen announced that the FDA agreed to review its experimental medicine for ALS even though a major clinical trial failed. Biogen is seeking approval based on the drug’s effect on a protein linked to neurological damage. Less than a month later, Brainstorm Cell Therapeutics said it would also seek FDA approval for an ALS drug after an additional analysis of a failed study revealed a statistical method error.
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