QUICKER APPROVALS FOR GENE THERAPIES? Peter Marks, the director of the FDA Center for Biologics Evaluation and Research, said the agency must more clearly define how it will use accelerated approval to advance the gene therapy field. The agency is using some funding from last year’s user fee reauthorization package to expand the number of staff assigned to cell and gene therapies, advance manufacturing technologies, study concurrent submission and review alongside other regulatory authorities and pilot an enhanced communication program for rare diseases, Marks told the Alliance for a Stronger FDA this week. “The hope is that with the combination of these different initiatives, we’ll be able to salvage some of what has happened in the small population gene therapy world with the downturn in the stock market and venture investment in this area,” Marks said. “A number of programs in industry that originally were looking at small populations have now been either given back to academics or just dropped entirely.” The goal, he said, is to reverse those trends and work toward a “commercially viable ecosystem.” But the agency competes with tech companies and the biopharmaceutical industry to hire reviewers, statisticians and epidemiologists — a challenge Marks says will be especially difficult this year. On the Covid-19 front: The agency’s top vaccine regulator said moving toward a simplified annual schedule for Covid vaccines should boost Americans’ vaccine confidence. “Part of that is probably communicating what [vaccines] can do — which is help save lives and prevent hospitalizations — and what they will not do, at least for this generation, which is prevent transmission or perhaps even prevent people from catching Covid-19,” Marks said. Looking ahead: If the U.S. does not implement a national infectious disease surveillance system capable of collecting real-time data on the spread of diseases like Covid, “we're going to be laughingstocks,” Marks added. PRETERM BIRTH DRUG PULLED — The FDA has formally withdrawn approval for Makena, a drug to help reduce the risk of preterm birth that had been on the market since 2011. Makena nor any of three generics can no longer be sold in the U.S., the agency said Thursday. The FDA had granted Makena accelerated approval, but in a post-marketing clinical trial that wrapped in 2019, the drug failed to prolong pregnancy in individuals susceptible to early labor nor did it benefit patients or babies. An independent advisory panel and an FDA official then recommended the drug be pulled from the market. Covis Pharma, Makena’s manufacturer, volunteered to pull the drug from the market in March. The FDA said it recognized that some supplies of the drug were already distributed and that patients should talk with their health care providers before taking the drug. “The touchstone of FDA drug approval is a favorable benefit-risk assessment; without that favorable assessment, the drug should not have the status of being FDA-approved,” FDA Commissioner Robert Califf said in a statement.
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