ADVISORY COMMITTEE NARROWLY VOTES AGAINST APPROVING ALS DRUG — Food and Drug Administration’s independent advisory committee on neurological drugs voted 4-6 against approving a drug intended to treat amyotrophic lateral sclerosis, or ALS, on Wednesday, Katherine reports. Committee members cited a need for more compelling data about the drug’s efficacy from a Phase III trial. Regulators from FDA pointed out shortcomings with the integrity of the data and the study design Amylyx submitted as part of its new drug application. The company included results from its Phase II randomized trial, called CENTAUR, and its open-label continuation. While committee members agreed the drug, called AMX0035, had no major safety concerns, they were skeptical of the metrics the company used to show that the drug modestly slowed the disease’s progression and extended patients’ lives. During the open public hearing, several patients living with ALS took the stand and repeatedly said they were eager for access to the drug — even if its benefits were small. Many committee members who voted against AMX0035’s approval were torn over their decision, noting the limited treatment options for ALS and the disease’s devastation. “Unfortunately, there are many features of the CENTAUR trial that limit its persuasiveness as a standalone trial in a regulatory sense,” said G. Caleb Alexander, a committee member who is a professor of epidemiology and medicine at Johns Hopkins Bloomberg School of Public Health. FDA is expected to make a final decision on the drug by June 29. Meanwhile, Amylyx is currently conducting a Phase III clinical trial, called PHOENIX, and plans to enroll 600 participants in the U.S. and Europe. The company estimates the study will end in 2024. MICHELE MITAL TO BECOME ACTING DIRECTOR OF FDA'S CENTER FOR TOBACCO PRODUCTS — Michele Mital will become the acting director of the Food and Drug Administration’s Center for Tobacco Products, Katherine reports. Mital is currently the deputy director of CTP; she’ll take over in an acting capacity for Mitch Zeller, who plans to leave the office this month. Mital has more than 25 years’ experience in FDA, including in the agency’s Office of Tobacco Programs, which predated the creation of the Center for Tobacco Products in 2009. “Michele is very strong operationally,” former FDA Commissioner Scott Gottlieb told POLITICO. “She’s going to be able to fully establish the institutional footprint they need for the long run.” CANDID CALIFF — FDA Commissioner Robert Califf weighed in on a number of top policy topics at STAT’s Breakthrough Science Summit on Thursday, including his rocky confirmation path earlier this year, FDA’s accelerated approval pathway and opioid policy. The longtime cardiologist, who recently returned to the top job at FDA after a stint at Alphabet, argued the commissioner's role is to work with the world outside the agency to put into place “systematic policies that would improve things” instead of weighing in on regulatory decisions. Califf argued that one area of needed improvement is creating an ecosystem where postmarket studies are efficiently completed after a drug is approved. “The commissioner is a political appointee, as well documented by confirmation exercise,” Califf quipped. “The people that make decisions about individual products are full-time civil servants who have no financial conflict of interest.” Califf indicated there might be a need to withdraw opioid products from the market or again change product labels in response to a question from STAT’s Matthew Herper. “We’re looking carefully at every option,” Califf said. “We can’t treat them like every other drug because of the horrendous addiction.” Combating misinformation is another top priority for Califf, who said there’s “a universe of digital information there all the time, much of which is completely incorrect.”
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