Table 2.
List of complement-related drugs without orphan designation in pre-clinical and clinical development for rare diseases.
| Compound Name | Manufacturer | Complement Target | Indication |
|---|---|---|---|
| Cetor C1 esterase inhibitor | Sanquin | C1r, C1s, MASP-1, MASP-2, other proteases | Hereditary Angioedema, Acquired C1-inhibitor deficiency |
| TNT009 Anti-C1s Antibody | True North Therapeutics | C1s | Cold Agglutinin Disease Phase l |
| Lampalizumab Anti-Factor D Antibody (FCFD4514S, RG7417) | Genentech | FD | Age-related Macular Degeneration Phase II/III |
| ACH-CFDIS Anti-Factor D inhibitor | Achillion | FD | Pre-clinical |
| CDX-1135 (TP10) soluble CR1 | Celldex | C3-convertase, C4b, C3b | Dense Deposit Disease Phase I |
| TT30 (ALXN1102) | Alexion Pharmaceuticals | C3-convertase, C3b | Paroxysmal Nocturnal Hemoglobinuria Phase I |
| TT32 | Alexion Pharmaceuticals | C3-convertase, C4b, C3b | Pre-clinical |
| Mini-FH | Amyndas | C3-convertase, C3b | Paroxysmal Nocturnal Hemoglobinuria Pre-clinical |
| Zimura (ARC1905) | Ophthotech | C5 | Age-related Macular Degeneration Phase II |
| LFG316 | Novartis | C5 | Dry Age-related Macular Degeneration Phase II |
| Coversin (OmCl) | Volution Immuno-Pharmaceuticals | C5 | Age-related Macular Degeneration Phase I |
| SOBI002 | Swedish Orphan Biovitrum | C5 | Phase I |
| RA101348 | Ra Pharmaceuticals | C5 | Pre-clinical |
| ALN-CC5 (Anti-C5 siRNA) | Alnylam | C5 | Paroxysmal Nocturnal Hemoglobinuria Phase I/II |
| CaCP29 (IFX-1) | InflaRx | C5a | Sepsis Phase II |