Duchenne Muscular Dystrophy Treatment Receives Pediatric Disease Status from FDA

Duchenne Muscular Dystrophy Treatment Receives Pediatric Disease Status from FDA

Marathon Pharmaceuticals, a company that develops new medications for rare diseases with a focus on patients who have no treatment options, recently announced that the U.S. Food and Drug Administration (FDA) has issued a rare pediatric disease designation to deflazacort to treat patients with Duchenne Muscular Dystrophy (DMD).

Deflazacort is an oxazoline derivative of prednisolone, a glucocorticoid recommended for anti-inflammatory diseases and typically used as an immunosuppressant. Although the drug is not approved in the United States, it has been commonly used in Europe and in Canada. According to a press release, the FDA’s decision was based on the results of clinical trials conducted in children with DMD that showed deflazacort, when compared to prednisone, was able to better conserve bone density with long-term use. In addition, DMD patients treated with the drug were less prone to develop scoliosis. The list of adverse side events experienced by study patients involved erythema, hirsutism, nasopharyngitis, cushingoid appearance, weight gain, irritability, and cataracts.

In August 2013, the FDA granted orphan drug designation for deflazacort as a treatment for this rare pediatric disease. In January 2015, the administration granted a fast track status to Marathon Pharmaceuticals to pursue approval of deflazacort as a potential treatment for Duchenne muscular dystrophy. According to the press release, the company is anticipating to submit a new drug application to the FDA for deflazacort in the first semester of 2016.

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Researchers at the University of Missouri have reported the complete and successful treatment of Duchenne muscular dystrophy (DMD) in dogs through injection of a viral vector carrying a dystrophin micro gene, leading to recovery of all muscles. Clinical trials involving humans are being planned as a next step for the team. The research paper, entitled “Safe and bodywide muscle transduction in young adult Duchenne muscular dystrophy dogs with adeno-associated virus,” was published in Human Molecular Genetics.

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