pnh

Iptacopan hydrochloride (LNP023 hydrochloride) is an orally bioavailable, highly potent and highly selective factor B inhibitor with an IC50 of 10 nM. Iptacopan hydrochloride shows direct, reversible, and high-affinity binding to human factor B with a KD of 7.9 nM.

Pelecopan (BCX9930) is a potent, selective, orally active inhibitor of complement factor D (IC50=14.3 nM) that prevents both intravascular and extravascular hemolysis in PNH.

Crovalimab (SKY59; RO7112689) is an Fc-engineered and pH-dependent humanised monoclonal antibody that binds to complement C5 and blocks its cleavage into C5a and C5b, thereby inhibiting MAC formation and intravascular haemolysis. It can rebind to the FcRn receptor under acidic pH conditions and return to the bloodstream, making it suitable for the treatment of paroxysmal nocturnal haemoglobinuria (PNH).

Danicopan (ACH-4471) (ACH-4471) is a selective, orally active small molecule factor D inhibitor with high binding affinity to human factor D, with a Kd value of 0.54 nM. Danicopan (ACH-4471) inhibits the activity of the complement replacement pathway (APC) and may block the complement replacement pathway in paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS).