US FDA's Record-Breaking Month Of Breakthrough Therapy Designations

The Council of the EU’s vision for the pharma reform package includes a stronger role for member states regarding the supply of medicines and the prevention of indication-stacking for orphan drugs.

Madrigal Pharmaceuticals’ Rezdiffra is on track to become the first medicine approved in the EU for non-cirrhotic metabolic dysfunction-associated steatohepatitis after the European Medicines Agency recommended that it be granted conditional marketing authorization.

The former CDER director said she tells sponsors not to conduct an FDA-recommended study design or randomized trial if it will not work.

Highlights from Day Three of the BIO International Convention include the realities of MFN pricing, AstraZeneca's R&D plans for obesity, the need for resources for the FDA's rare disease hub and reactions to the Commissioner's National Priority Review Voucher program.