The company had pivoted towards a drug with the opposite mechanism to Novo Nordisk’s Wegovy, but ran out of time and money.

Immuno-oncology will remain a major force at both FDA’s drugs and biologics centers, but watch for psychiatry and antibiotics to make some noise; rare pediatric diseases will remain prominent as the priority review voucher program heads into the sunset.

Last year, the European Commission approved a record 55 therapies containing a new active substance, including 21 medicines for rare diseases. Among them were a number of gene therapies for indications such as cancer and hemophilia.

Despite broad interest, real-world evidence of efficacy in FDA decisions remains largely limited to orphan diseases and natural history, a Pink Sheet analysis shows.