Sanders, Cummings Announce Congressional Investigation into Marathon’s Recently Approved Emflaza® (deflazacort)

On February 9, FDA announced approval of a new drug to treat Duchenne muscular dystrophy. In fact, NDAs 208684 and 20685 (oral tablets 6, 18, 30, and 36 mg and oral suspension 22.75 mg/mL), both orphan drugs, were approved that day.  However, by February 13, Reuters reported that “Marathon pauses Duchenne drug launch amid price outcry”.  Today (February 21), FDAnews Drug Daily Bulletin reported that “Sanders, Cummings Launch Congressional Investigation into Marathon’s $89,000 Drug”.  Click here for the brief but full story.  Click here to request assistance with orphan drug designation and/or orphan pricing guidance.