Amylyx Pharmaceuticals' experimental drug has become a rallying cause for patients with Lou Gehrig’s disease, their families and members of Congress who are pushing the FDA to approve the drug.
This 2018 photo provided by Amylyx shows the company's co-founders Joshua Cohen, left, and Justin Klee in Cambridge, Mass. On Friday, Sept. 2, 2022, federal health regulators remain unconvinced about the benefits of a closely watched experimental drug for the debilitating illness known as Lou Gehrig’s disease, even as they're set to give its maker, Amylyx, a rare second chance to make its case publicly.
Elsewhere in its review the FDA detailed the flexibility it can apply to drug approval decisions, particularly for deadly diseases, which suggests “there is a chance that the FDA is still looking for a way to approve the product,” SVB analyst Marc Goodman wrote in a note to investors. He gives Amylyx a 50% chance of approval.
Amylyx’s data comes from one small, mid-stage trial that showed some benefit in slowing the disease, but which was marred by missing data, implementation errors and other problems,Amylyx says follow-up data gathered after the study concluded shows the drug extended life. When the company followed patients who continued taking the drug, they survived about 10 months longer than patients who never took the drug, according to a new company analysis.
Amylyx’s drug is a combination of two older drug ingredients: a prescription medication for liver disorders and a dietary supplement associated with traditional Chinese medicine. The Cambridge, Massachusetts, company has patented the combination and says the chemicals work together to shield cells from premature death. Its co-founders first hit upon the combination as Brown University students.
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