Arrowhead’s in-house, late-stage cardiovascular disease programs will center on triglyceride-lowering plozasiran while it seeks a partner to fund Phase III for cholesterol-reducing zodasiran.

Sometimes even the US FDA’s regulatory flexibility may not be enough to approve rare disease treatments in very small populations, but agency officials worry that creating a new pathway could be problematic.

The company expects to have data from transfusion-dependent and non-transfusion dependent patients this year, with a potential expanded US approval in 2025 for Pyrukynd.

Vertex and bluebird compete for sickle cell patients, BioMarin talks about divesting Roctavian, and Sarepta warned that sales of Elevidys will plateau with a narrow label.