U.S approval of orphan drug Erwinase puts specialty pharma firm EUSA on new growth trajectory, showing the value of its strategy to build a transatlantic infrastructure for niche products – and transitioning it from a commercially-focused to a development-capable organization.

The National Organization for Rare Disorders’ campaign to get FDA to codify a flexible approach on approving orphan drugs is now bolstered by an analysis that concludes most of the agency’s approval decisions for such products already use unconventional efficacy standards.

FDA reviewers stuck to established assessment practices during the coronavirus crisis and produced one of the agency’s largest yearly novel approval counts ever.