Minoryx Preps For Tough Pricing Regime As CNS Orphan Drug Enters Phase II/III
Executive Summary
Orphan drug development used to be considered a license to print money, with high price tags and a focused customer base. With the development of an increasingly tough orphan drug pricing environment in the US and Europe, biotechs are adopting new strategies. CNS drug developer Minoryx will introduce endpoints into its Phase II/III studies for rare brain disease X-ALD that should help demonstrate both efficacy and value for future pricing discussions.