- Based on a positive pre-NDA meeting with the FDA, privately-held Marathon Pharmaceuticals begins the New Drug Application process seeking approval for Fast Track- and Orphan Drug-tagged deflazacort (MP-104) for the potential treatment of Duchenne muscular dystrophy (DMD).
- The company expects to submit its NDA in Q1 2016. If all goes well, marketing clearance could happen in Q1 2017, although it can request that the FDA designate it for Priority Review which would shorten the clock to six months from 10.
- Marathon also seeks authorization from the FDA to provide deflazacort at no charge as an investigational drug to qualified DMD patients via an expanded access program. The agency stated that it may formally authorize the program. A decision could happen in August or September.
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Deflazacort is a glucocorticoid with anti-inflammatory and immunosuppressive properties. It is not currently available in the U.S. but is approved for a variety of non-DMD indications ex-U.S.
- DMD-related tickers: (FGEN +0.5%)(PFE)(SRPT +2.5%) (NASDAQ:RNA) (PTCT +1.5%)(MRNA +1.4%)(CAPR -3.5%)(OTCPK:SPHDF)(BMRN +1%)(SMMT -2.2%)