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Marathon Pharmaceuticals (officially Marathon Pharmaceuticals, LLC) was a privately held pharmaceutical company based in Northbrook, Illinois, focused on rare diseases. Its most notable achievement was the FDA approval and commercialization of deflazacort (branded as Emflaza) for Duchenne muscular dystrophy (DMD), a fatal pediatric neuromuscular disorder. Marathon's strategy centered on repurposing established generic drugs—such as deflazacort, a synthetic glucocorticoid—gaining FDA exclusivity via the Orphan Drug Act, and setting high US prices, which generated significant controversy.
The company exited the market following public and political backlash over drug pricing, selling its deflazacort assets to PTC Therapeutics for approximately $140 million in 2017. Marathon is not currently active as an independent entity, and its drug development pipeline was limited, with several programs discontinued. Its legacy is as a case study in the exploitation of regulatory incentives for rare disease drugs and the ethical debates surrounding pharmaceutical pricing.
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