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James Graham Brown Cancer Center
Division of University of Louisville
Latest From Marathon Pharmaceuticals LLC
Repeated trial failures in this rare disease space have given birth to new alliances – one backed by Critical Path and the other by the CureDuchenne advocacy group – that aim to model disease progression much more accurately through data sharing and rigorous analysis.
Wall Street Journal brings Marathon Pharmaceuticals back to the front pages – but this time to report that the company is expected to shut down after a 'drug-price revolt.' The rest of the industry should hope the headline catches the President’s eye.
Filing supporting Brineura was supported by a tiny, single-arm study and had no advisory committee review, the latest sign of the US regulatory agency's willingness to speed orphan drugs for serious pediatric diseases.
With BioMarin's Brineura hitting the market with a $702,000 annual price tag, recent experience for other high-priced rare disease drugs supports the theory that payers will accept high costs for ultra-rare pediatric disease therapies.
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