Asklepion Pharmaceuticals, LLC

The exigencies of ultra-orphan drug development have provided the early testing ground for the US FDA’s limited use of real-world evidence (RWE) in approval decisions, resulting in 13 approvals identi

The pricing controversy surrounding Marathon Pharmaceuticals LLC 's aborted launch of Emflaza (deflazacort) for Duchenne muscular dystrophy has also drawn critical attention to what Sen. Bernie S

Six months after emerging from bankruptcy, KaloBios Pharmaceuticals Inc. announced that it is on track to file a 505(b)(2) new drug application for its lead compound benznidazole for the treatment

In a judgment that upholds a central tenet of Europe's Orphan Drug Regulation, the Court of Justice of the EU has ruled that all orphan medicines are entitled to a full 10-year period of market exclus