Also known as: Emflaza
Emflaza is the brand name for deflazacort oral formulations, a corticosteroid indicated for Duchenne muscular dystrophy in patients aged 2 years and older. It is dosed once daily and requires tapering if used beyond a few days. Initial U.S. approval occurred in 2017.
PTC Therapeutics has withdrawn its New Drug Application resubmission for ataluren (Translarna) for nonsense mutation Duchenne muscular dystrophy after the FDA indicated the submission lacked substantial evidence of effectiveness.
PTC Therapeutics has withdrawn its New Drug Application for Translarna (ataluren) for Duchenne muscular dystrophy after the FDA indicated the data were unlikely to meet approval thresholds, ending a pursuit spanning more than a decade.
