Sino-US biotech Kira Pharmaceuticals announced that it has received an Orphan Drug Designation (ODD) from the US Food and Drug Administration (FDA) for KP104, its first-in-class biologic for the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH).
Drug Profile
KP104 is a bifunctional biologic designed to selectively block the alternative and terminal pathways of the complement system, addressing the root cause of PNH. The drug’s unique dual-approach mechanism provides a synergistic method to target disease drivers. KP104 is engineered for extended half-life and potency and can be administered via IV or subcutaneous routes.
Clinical Trials
KP104 is advancing into Phase II trials for multiple indications, including IgA nephropathy (IgAN), C3 glomerulopathy (C3G), thrombotic microangiopathies secondary to systemic lupus erythematosus (SLE-TMA), and PNH. Trials will be conducted globally, with sites in the US, China, Australia, and South Korea.-Fineline Info & Tech
