PTC wins US approval of gene therapy for fatal enzyme disorder

Kebilidi is the first approved gene therapy that can be directly administered to the brain. Its OK secures a priority review voucher for PTC.

Syndax secures FDA OK for new kind of leukemia drug

Revuforj is the first so-called menin inhibitor cleared in the U.S., securing FDA approval for use in adults and certain pediatric patients with an aggressive form of acute leukemia.

A look inside the latest advancements in cell therapy

Eisai wins over European regulators on Alzheimer’s drug Leqembi

The EMA sided against the drug this summer. But an appeal from Eisai appears to have worked, teeing Leqembi up for authorization in a major market.

Astellas application rejected by FDA; Cytokinetics strikes licensing deal with Bayer

The agency turned back Astellas’ attempt to update its drug Izervay’s labeling. Elsewhere, former NCI director Ned Sharpless founded a new startup and Novartis licensed another radiopharma drug.

Novo Nordisk launches obesity drug Wegovy in China