Another old drug looks for a second coming at the FDA, this time for Duchenne MD
So far, drugs that try to address the root cause of Duchenne muscular dystrophy have had to travel a rocky road at the FDA. But regulators are now hustling up a review of a new treatment — new to the US at least— that will try to do what a lineup of well financed biotechs and pharma companies have failed at.
Marathon Pharmaceuticals put their DMD candidate deflazacort through a Phase III study, and it says the drug provided evidence of improving the muscle strength of boys, letting them do better at a variety of physical tasks, like sitting up or walking. On Wednesday, the FDA provided priority review status for the drug when it accepted the application for review, setting up an accelerated February PDUFA deadline on whether it should be used to treat DMD boys with the lethal disease.
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