Terminated trial offered continued access to rare blood disorder drug
Disease control
Terminated
This study provided continued access to the experimental drug BCX9930 for 28 people with paroxysmal nocturnal hemoglobinuria (PNH), a rare and serious blood disorder. Participants had previously benefited from BCX9930 in earlier studies and didn't have other treatment options. Th…
Phase: PHASE2 • Sponsor: BioCryst Pharmaceuticals • Aim: Disease control
Last updated Mar 30, 2026 14:30 UTC