The FDA on Friday revised the safety information for Sarepta Therapeutics’ Duchenne muscular dystrophy gene therapy, Elevidys, adding a boxed warning to alert patients and doctors about the risk of severe liver damage and possible death. This highest-level warning outlines the chance of serious liver injury and acute liver failure in people who receive Elevidys, with both complications carrying the possibility of being fatal. The agency has also limited the therapy’s use to ambulatory Duchenne patients who are at least 4 years old and have a confirmed mutation in the DMD gene. With these changes, Elevidys is no longer permitted for patients who cannot walk.What Is Duchenne Muscular Dystrophy?Duchenne muscular dystrophy (DMD) is a serious inherited condition that leads to steady muscle weakening and loss, affecting boys far more often. It stems from a defect in the dystrophin gene, which prevents the body from making a key protein that protects muscle fibers. Signs usually show up in early childhood and include trouble walking or climbing, frequent falls and a noticeable waddling walk, according to the Cleveland Clinic.What Is The Duchenne Gene Therapy?Gene therapy for Duchenne muscular dystrophy delivers a corrected or shortened form of the dystrophin gene into muscle cells using a modified virus. This allows the cells to produce a working version of the protein. The single intravenous infusion is designed to slow the disease by restoring some dystrophin, with the aim of improving movement and daily functioning. While the FDA has cleared a therapy that creates a reduced “micro-dystrophin,” researchers are still working on ways to provide the full-length gene for potentially stronger results, as noted by the Food and Drug Administration.What Is Elevidys?Elevidys is a gene-based treatment designed for Duchenne muscular dystrophy. It is given only once and relies on an adeno-associated virus to carry a modified form of the dystrophin gene into the body. This shorter micro-dystrophin version is meant to take the place of the faulty or absent dystrophin that leads to the condition.FDA Tightens Duchenne Gene Therapy After Deaths Of Two TeensThe U.S. Food and Drug Administration is tightening control over a Duchenne muscular dystrophy gene therapy after two teenagers died from liver failure linked to the treatment. The agency has restricted Elevidys, made by Sarepta Therapeutics, to boys aged 4 and older who are still able to walk. It can no longer be given to boys who have already lost the ability to move independently, something that usually occurs around age 12 in Duchenne. A safety review confirmed that both teenagers developed severe liver damage after receiving the infusion and later died.The FDA is also adding its strongest boxed warning to call attention to the risks of serious liver injury, acute liver failure and death. Sarepta, based in Cambridge, Massachusetts, with offices in several countries, told regulators earlier this year that it had already stopped supplying the therapy to non-ambulatory patients. The company stated that Elevidys has been used in 1,100 patients worldwide.These updates “will ensure that families and health-care professionals have clear information” to support treatment choices, Louise Rodino-Klapac, Sarepta’s head of research and development, told The New York Times.The FDA’s new label comes at the end of a difficult year for Sarepta, marked by weak clinical trial results, safety questions surrounding Elevidys and declining sales.
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