Marathon's High-Priced Window May Not Stay Open Long
Ongoing trial is intended to compare the newly approved steroid Emflaza with prednisone in DMD for efficacy and safety.
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Pink Sheet's monthly Drug Review Profile looks at Marathon's (now PTC's) Emflaza for Duchenne muscular dystrophy, and finds that before the steroid's price ignited controversy it received an unremarkable review – unremarkable, that is, for rare and serious illnesses. The US FDA accepted old and flawed trial data because signals of clinical benefit were consistently seen and safety profile was understood.
House bill that would require new clinical studies from sponsor for tropical disease priority review vouchers met with industry concerns about limiting the US agency's flexibility.
Can a company explain the situation surrounding a newly approved drug, including the effect of the indication on access, without violating off-label communication regulations?