Table 1: An overview of (pre)clinical complement inhibitors.

Complement inhibitorMedicineDiseasesStudy phase

Recombinant C1 inhibitor Conestat alfaHAE
Side effects: headache and allergy.
In clinical use, EU approved.
(Ruconest in Europe/Rhucin in USA)

Plasma-derived C1 inhibitorsBerinert P/cinryzeHAEIn clinical use, FDA approved.

C3 inhibitorsCompstatin (POT-4)AMDPhase II
Staphylococcal complement inhibitor (SCIN)Preclinical

Myristoylated peptidyl derived from soluble CR1Mirococept (APT070)Delayed graft function of cadaveric kidney after transplantation. Phase II

Factor HPlasma-derived factor H concentrate HUS, AMD Preclinical
TT30/targeted alternative pathway inhibitor/factor HPNH, AMDPhase I

Factor D inhibitorAnticomplement factor DAMDPhase II

Factor B inhibitorTA106/anti-complement factor BAMDPreclinical

C5 inhibitorsEculizumabPNH
Side effects: headache, thrombocytopenia, gastrointestinal complaints and infections.
Before use: vaccination against meningococcal infection.
In clinical use, FDA approved.
Various other diseases, for example, kidney transplants, HUS, AMD.Phase I
PexelizumabPhase III study failed
ErgidinaIschemia/reperfusion injuryPreclinical
ARC 1905AMDPhase I

C5a inhibitorPMX 53 and several other compoundsAMDPhase II study discontinued
OsteoarthritisPhase I

Targeted complement inhibitorsTargeted
(CR2 mediated) complement inhibitors
Chronic glomerulonephritisPhase I

HAE: hereditary angioedema; AMD: acute macular degeneration; HUS: haemolytic uraemic syndrome; PNH: paroxysmal nocturnal haematuria.