… We have to manage through the uncertainty here.” Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, said his agency will take the recommendation and “do something that we have to do every day at FDA. “The downside of the gene therapy here is relatively small compared to whether it really helps the patient, and for this reason, I voted yes,” he said.ĭr. Raymond Roos, a neurology professor at the University of Chicago Medical Center, voted in favor. Linda Wang/UCLA Broad Stem Cell Research CenterĪ drug company abandoned a treatment for 'bubble boy disease.' After a 5-year fight, this little girl is about to get itĭr. “The totality of evidence … simply doesn’t rise to the threshold that’s required for accelerated approval.” Caleb Alexander, a professor of epidemiology and medicine in the Bloomberg School of Public Health at Johns Hopkins University, who voted against recommending approval. “The decision the FDA has to make doesn’t just affect the patients in study 301 it affects the entire field of drug development for Duchenne,” said Dr. The advisers voted 8-6 in favor of approving the treatment, and the FDA will now decide whether to follow their advice. Hennick and many other parents like her advocated for the treatment’s accelerated approval Friday in a meeting of outside advisers to the US Food and Drug Administration. SRP-9001 uses viruses to ferry a copy of a gene to muscles to help make up for one that’s causing the disease. Current treatments for the disease – which primarily affects boys because of the way it’s inherited – include steroids and, later, heart drugs. HennickĬonnor is one of just dozens of kids to have received SRP-9001, an experimental gene therapy that aims to slow or stop the progression of Duchenne muscular dystrophy, or DMD. Connor Hennick was admitted to the gene therapy trial at age 7.
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