US FDA contemplating how to match orphan designation process with drug development moving to molecularly-targeted therapies.
Merck & Co. Inc.'s new molecularly-targeted indication for Keytruda could help open the door to orphan incentives for such products.
US FDA Commissioner Scott Gottlieb said discussions are ongoing inside the agency about whether an indication based on a genetic...
While treatments for scleroderma are in development, none are approved for the condition that is associated with high morbidity and mortality. The European Medicines Agency says guidance on how to design development programs for the disorder is needed.
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.
Highlights from Day Four of the BIO International Convention include policy concerns helping constrain dealmaking, Novartis discussing its approach to partnering, and Generate looking for funding to move into Phase III.
