The Food and Drug Administration (FDA) has granted Orphan Drug designation to KP104 for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). The FDA’s Orphan Drug designation is granted to medicines intended to treat or prevent rare diseases or disorders that affect fewer than 200,000 individuals.

KP104 is a bifunctional antibody fusion protein that works by selectively inhibiting the alternative and terminal pathways in the complement system. The investigational therapy is engineered to have an extended half-life and potency, with a formulation suitable for both intravenous and subcutaneous administration.

The Company has completed a phase 1 dose-ranging clinical trial in healthy volunteers. Study data is expected to be presented at a medical conference later this year.

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“As a bifunctional complement therapy targeting both the alternative and terminal pathways, KP104 has the potential to treat complement-mediated diseases where single-target therapies are not adequate,” said Frederick Beddingfield, MD, PhD, CEO at Kira Pharmaceuticals. “As we enter phase 2 across multiple indications, we look forward to continued clinical evaluation of KP104 for patients in need.”

Kira plans to also evaluate KP104 for IgA nephropathy, C3 glomerulopathy, and thrombotic microangiopathies secondary to systemic lupus erythematosus.


Kira Pharmaceuticals receives FDA Orphan Drug designation for KP104, a bifunctional antibody fusion protein, for the treatment of paroxysmal nocturnal hemoglobinuria. News release. Kira Pharmaceuticals. Accessed July 28, 2022.

This article originally appeared on MPR