Drug discovery
14
drugs
With orphan designations
Overview
C3 glomerulopathy (C3G) is a rare complement dysregulation disorder driven by alternative pathway overactivation, characterized by C3 protein deposition in glomeruli. It encompasses dense deposit disease (DDD) and C3 glomerulonephritis (C3GN), diagnosed via kidney biopsy. Chronic progression leads to kidney damage in ~50% of patients within 10 years, with high recurrence rates post-transplant. No disease-modifying therapies exist, necessitating supportive care and targeted clinical trials [1][3][9].
Therapies
First-line: ACE inhibitors/ARBs for proteinuria; renoprotective measures (SGLT2 inhibitors, dietary sodium/protein restriction) [2][3][13]
Immunomodulation: Mycophenolate mofetil + glucocorticoids for moderate-severe disease (36-65% remission rate) [2][8][17]
Emerging therapies: Anti-C5 (eculizumab), factor B inhibitors (iptacopan), C3-targeted drugs (pegcetacoplan) in late-stage trials [3][9][17]
Categories: rare genetic diseases, rare renal diseases, rare transplant-related disorders
Drug Discovery Landscape
Drug | Therapy type | Regulator | Orphan designation | Approval | Sponsor |
|---|---|---|---|---|---|
chemically synthesized double-stranded oligonucleotide directed against C3 messenger RNA | oligonucleotides | FDA | 2025-10-16 | — | Sanegene Bio USA Inc. |
Complement factor H, human, recombinant | proteins | EMA | 2024-06-28 | — | Greenovation Biotech GmbH |
synthetic double-strand small interfering ribonucleic acid (siRNA) oligonucleotide drug directed against C3 messenger RNA | RNAs | FDA | 2022-11-22 | — | Silence Therapeutics plc |
Pegcetacoplan [ASPAVELI] | peptides | EMA | 2022-11-10 | 2026-01-16 | Swedish Orphan Biovitrum AB (publ) |
iptacopan [Fabhalta] | small molecules | FDA | 2020-05-18 | 2025-03-20 | Novartis Pharmaceuticals Corporation |
Poly(oxy-1,2-ethanediyl), alpha-hydro-omega-hydroxy-,15,15'-diester with N-acetyl-L-isoleucyl-L-cysteinyl-L-valyl-1-methyl-L-tryptophyl-L-glutaminyl-L-alpha-aspartyl-L-tryptophylglycyl-L-alanyl-L-histidyl-L-arginyl-L-cysteinyl-L-threonyl-2-[2-(2-aminoethoxy)ethoxy]acetyl-N6-carboxy-L-lysinamide cyclic (2.fwdarw.12)-(disulfide); where two identical synthetic peptide domains are covalently linked at the ends of the polyethylene glycol chain | peptides | EMA | 2019-08-21 | — | Swedish Orphan Biovitrum AB (publ) |
pegcetacoplan [Empaveli] | peptides | FDA | 2018-12-19 | 2025-07-28 | Apellis Pharmaceuticals, Inc. |
(4-{(2S,4S)-4-ethoxy-1-[(5-methoxy-7-methyl-1H-indol-4-yl)methyl]piperidin-2-yl}benzoic acid-hydrogen chloride(1/1)) [FABHALTA] | small molecules | EMA | 2018-12-14 | 2025-04-01 | Novartis Europharm Limited |
Danicopan | small molecules | EMA | 2018-03-21 | — | Alexion Europe |
(2S,4R)-1-(2-(3-acetyl-5-(2-methylpyrimidin-5-yl)-1H-indazol-1-yl)acetyl)-N-(6-bromopyridin-2-yl)-4-fluoropyrrolidine-2-carboxamide | small molecules | FDA | 2017-12-14 | — | Alexion Pharmaceuticals, Inc |
Avacopan | small molecules | EMA | 2017-06-20 | — | Chemocentryx Ireland Limited |
avacopan | small molecules | FDA | 2017-03-20 | — | ChemoCentryx, Inc. |
S3,S13-cyclo(D-tyrolsyl-L-isoleucyl-L-cysteinyl-L-valyl-1-methyl-L-tryptophyl-L-glutaminyl-L-aspartyl-L-tryptophyl-N-methyl-L-glycyl-L-alanyl-L-histidyl-L-arginyl-L-cysteinyl-N-methyl-L-isoleucinamide) | peptides | EMA | 2016-02-17 | — | Amyndas Pharmaceuticals S.A. |
S3,S13-cyclo(D-tyrolsyl-L-isoleucyl-L-cysteinyl-L-valyl-1-methyl-L-tryptophyl-L-glutaminyl-L-aspartyl-L-tryptophyl-N-methyl-L-glycyl-L-alanyl-L-histidyl-L-arginyl-L-cysteinyl-N-methyl-L-isoleucinamide | peptides | FDA | 2015-11-16 | — | Amyndas Pharmaceuticals S.A |