A Phase III hit is always nice but a lot more information will be required to judge iptacopan’s market potential.

US FDA lauds collaborative development of surrogate endpoint that allowed for accelerated approval of the first two drugs for the rare kidney disease. The program was presented as an example of novel endpoints for rare diseases at a Duke/FDA workshop.

Travere’s drug, approved as Filspari for immunoglobulin A nephropathy, missed its eGFR primary endpoints in focal segmental glomerulosclerosis but showed meaningful improvement in proteinuria.