IN A 180 MOVE, EXTERNAL ADVISERS ENDORSE ALS DRUG — The FDA’s advisory committee on neurological drugs voted 7 to 2 Wednesday in favor of recommending the agency approve a novel amyotrophic lateral sclerosis drug candidate from Amylyx Pharma, your host reports. Déjà vu: The meeting was the second time the FDA convened an expert panel about the drug candidate. In March, advisers voted against the drug’s approval, citing concerns with the evidence generated from a single Phase II study. What changed: The company included two new analyses of its Phase II study and data from a Phase II trial of the drug on Alzheimer’s patients, which demonstrated the drug’s ability to improve biomarkers associated with neurodegeneration. Some advisers criticized this data rehashing, which they viewed as moving goalposts. “There’s a reason that in games and sports … the rules are decided before the game, not after the game, not after the plays are made,” said G. Caleb Alexander, a professor of epidemiology and medicine at the Johns Hopkins Bloomberg School of Public Health Center for Drug Safety and Effectiveness, who voted no against a favorable recommendation. But others pointed to the severe unmet need for ALS drugs, which could help make the case for the FDA to use its regulatory flexibility. The two other drugs previously approved for ALS, which both treat the symptoms like Amylyx’s candidate does, were brought to market with different evidence than is usually required. Plus, Amylyx’s candidate has an ongoing Phase III clinical trial, which advisers saw as a way to assess the drug’s future success. If results, expected in a few years, are positive, patients will have access to the drug anyway; if not, Billy Dunn, the head of the agency’s Office of Neuroscience in the Center for Drug Evaluation and Research, noted that the FDA could pull the drug from the market. Eyes emoji: “If PHOENIX [the Phase III trial] is not successful, we will do what is right for patients, which includes voluntarily removing the product from the market,” said Justin Klee, the co-founder and co-CEO of Amylyx. The FDA is expected to make a final decision on Amylyx’s application by Sept. 29. FDA APPROVES BOTOX ALTERNATIVE — The FDA approved Daxxify, a new anti-wrinkle injection from the biotech company Revance, on Wednesday. Like Botox, the 20-year-old drug from AbbVie that now makes up about 70 percent of the facial injection market, Daxxify uses a botulinum toxin to freeze facial muscles. But Revance is banking on Daxxify’s longer-lasting effects — up to six months instead of three to four — to grab a share of the multibillion-dollar space.
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