C5 complement C5
ENSG00000106804 · protein coding · Open Targets profile
Precursor of the C5a anaphylatoxin and complement C5b components of the complement pathways, which consist in a cascade of proteins that leads to phagocytosis and breakdown of pathogens and signaling that strengthens the adaptive immune system (PubMed:12878586, PubMed:18204047, PubMed:30643019, PubMed:6554279). Activated downstream of classical, alternative, lectin and GZMK complement pathways (PubMed:12878586, PubMed:18204047, PubMed:30643019, PubMed:39914456, PubMed:39814882, PubMed:6554279). {ECO:0000269|PubMed:12878586, ECO:0000269|PubMed:18204047, ECO:0000269|PubMed:30643019, ECO:0000269|PubMed:39814882, ECO:0000269|PubMed:39914456, ECO:0000269|PubMed:6554279}.
Each band is a group of drugs. It runs from the kind of drug on the left to the furthest stage that drug has reached in any disease on the right. Thicker bands mean more drugs.
Approved bands are highlighted. Withdrawn means the drug was pulled from the market or from development. A combined phase counts as the later one (Phase 1/2 is shown as Phase 2). Hover a band for the drug names. Source: Open Targets Platform, fetched live.
Each row is a cancer, strongest overall evidence first. The two dots show how strong the inherited (germline) and tumour-mutation (somatic) evidence is; the line between them is the gap.
Scores run from 0 (no evidence) to 1 (strongest). A missing dot at 0 means Open Targets holds no evidence of that kind. Source: Open Targets Platform, fetched live.
Tractability
- Small molecule
- Structure with Ligand, High-Quality Ligand, Med-Quality Pocket
- Antibody
- Approved Drug, UniProt loc high conf, GO CC high conf, UniProt SigP or TMHMM
- PROTAC
- Database Ubiquitination, Half-life Data, Small Molecule Binder
- Other modalities
- Approved Drug
gnomAD loss of function constraint: o/e 0.6399000287055969 (upper bound 0.736270010471344). Low values mean loss of function is poorly tolerated, a safety flag for inhibition.
Who runs trials of these drugs
Principal investigators and study chairs on 129 registry trials, updated in the last three years, of the most advanced drugs against C5 (vilobelimab, zilucoplan, avacincaptad pegol, crovalimab, pozelimab, eculizumab, ravulizumab, pexelizumab, nomacopan, cemdisiran, olendalizumab, tesidolumab), ranked by Phase 2+ trials. Live from ClinicalTrials.gov.
- UCB Cares001 844 599 2273 · 6 of 8 trials Phase 2+
- Alex GO Loayzauniversity · 1 of 1 trials Phase 2+
- Arnaud GarnierUniversity Hospital, Toulouse · 1 of 1 trials Phase 2+
- Austin G KulasekararajKing's College Hospital NHS Trust · 1 of 1 trials Phase 2+
- Christophe LEGENDREService de Transplantation Rénale, Hôpital Necker Université Paris Descartes 149 rue de Sèvres 75015 Paris, France · 1 of 1 trials Phase 2+
- Dr. D. SchadendorfUniversity Hospital, Essen · 1 of 1 trials Phase 2+
- Edward PhilpotOmeros Corporation · 1 of 1 trials Phase 2+
- Fadi FAKHOURINantes CHU · 1 of 1 trials Phase 2+
- Karine BrochardUniversity Hospital, Toulouse · 1 of 1 trials Phase 2+
- Maria Lourdes Gonzalez SuarezMayo Clinic · 1 of 1 trials Phase 2+
- Merit CudkowiczMassachusetts General Hospital · 1 of 1 trials Phase 2+
- Morag GriffinLeeds Teaching Hospitals NHS Trust, Leeds, UK · 1 of 1 trials Phase 2+
- Philippe-Antoine BilodeauMassachusetts General Hospital · 1 of 1 trials Phase 2+
- Richard BurwickCedars-Sinai Medical Center · 1 of 1 trials Phase 2+
- Rong FuTianjin Medical University General Hospital · 1 of 1 trials Phase 2+
Registry trials of vilobelimab, zilucoplan, avacincaptad pegol, crovalimab, pozelimab, eculizumab, ravulizumab, pexelizumab, nomacopan, cemdisiran, olendalizumab, tesidolumab updated in the last three years, counted by the country group of their listed sites. A trial with sites in several groups counts in each; 58 of 123 have all sites in one country.
Site counts are not patients per country, and trials registered only outside ClinicalTrials.gov are not covered. Where sites are is a fact about the record, not a judgement of its quality. Europe is the EU and EEA, the UK and Switzerland. 6 trials list no sites and are left out. Source: ClinicalTrials.gov, fetched live.
44 trials of drugs against C5 were stopped early; 2 give no reason
Terminated, withdrawn and suspended interventional trials of vilobelimab, zilucoplan, avacincaptad pegol, crovalimab, pozelimab, eculizumab, ravulizumab, pexelizumab, nomacopan, cemdisiran, olendalizumab, tesidolumab, newest update first, live from ClinicalTrials.gov. Each reason is quoted exactly as the registry gives it; we do not sort reasons into safety, efficacy or business. Dates are the last registry update. Showing the 25 most recent of 44.
- terminated · 2026-11-10 · BioCryst Pharmaceuticals · Eculizumab, Ravulizumab“Sponsor decision”
- terminated · 2026-11-04 · Hoffmann-La Roche · matched by the registry's drug synonyms“The study was terminated prematurely by the Sponsor due to observation of dose-limiting adverse events meeting the protocol-defined stopping criteria.”
- terminated · 2026-10-29 · Alexion Pharmaceuticals, Inc. · Ravulizumab“The ALXN1210-CSA-AKI-318 trial was discontinued on 29-Apr-2026 due to lack of efficacy in this overall study universe / patient population.”
- withdrawn · 2026-06-09 · University Hospital, Rouen · Eculizumab administration“Change in the molecule”
- withdrawn · 2026-03-02 · Hoffmann-La Roche · Crovalimab“The study was discontinued due to a strategic business/portfolio decision by the sponsor and was not related to any medical or safety issue.”
- terminated · 2026-01-22 · Alexion Pharmaceuticals, Inc. · Ravulizumab“Sponsor Decision.”
- withdrawn · 2025-11-20 · Assistance Publique - Hôpitaux de Paris · Eculizumab“disengagement of the sponsor Alexion Pharmaceuticals”
- terminated · 2025-11-04 · InflaRx GmbH · vilobelimab“Study stopped for futility”
- withdrawn · 2025-09-10 · Tianjin Medical University General Hospital · matched by the registry's drug synonyms“The study plan need to be modified after widely discussion with our consultation board, regarding the conscern about the efficiency may not be able to detect the expected differences. We would like to withdraw this study plan and startup a new one.”
- terminated · 2025-07-09 · Alexion Pharmaceuticals, Inc. · Ravulizumab“Sponsor decision”
- terminated · 2025-06-05 · AKARI Therapeutics · nomacopan (rVA576)“The early termination of this study is a business decision following a portfolio reprioritization plan. The decision is not related to any Efficacy, Safety or Clinical concerns regarding Nomacopan/rVA576”
- terminated · 2025-04-13 · BioCryst Pharmaceuticals · Eculizumab, Ravulizumab“Sponsor decision”
- terminated · 2025-04-10 · AKARI Therapeutics · matched by the registry's drug synonyms“The early termination of this study is a business decision, Akari have made the decision to close their global Phase III PNH program. The decision was not related to any efficacy, safety or clinical concerns regarding Coversin/rVA576.”
- terminated · 2025-04-10 · AKARI Therapeutics · matched by the registry's drug synonyms“The trial has terminated early due to the disruptions caused by the ongoing COVID-19 pandemic. The decision was not related to any efficacy, safety or clinical concerns regarding rVA576.”
- withdrawn · 2025-04-09 · AKARI Therapeutics · nomacopan (rVA576)“Akari has decided to discontinue AK802 study due to strategic resource allocation decisions.”
- terminated · 2025-04-08 · Regeneron Pharmaceuticals · Cemdisiran, Eculizumab, Pozelimab, Ravulizumab“Sponsor decision not related to efficacy or safety.”
- terminated · 2025-03-13 · IVERIC bio, Inc. · Avacincaptad Pegol“Following Sponsor reassessment, OPH2004 enrollment was stopped to initiate a new Zimura wet AMD trial, OPH2007, for treatment-naïve patients. Thus, only one patient was enrolled into and completed Study OPH2004.”
- terminated · 2025-02-11 · InflaRx GmbH · Vilobelimab, Vilobelimab + pembrolizumab combination therapy“Sponsor decided to stop the development of vilobelimab in cSCC and early terminated the IFX-1-P2.8 trial due to new alternative treatments for cSCC with high efficacy rates.”
- terminated · 2024-11-20 · Alexion Pharmaceuticals, Inc. · matched by the registry's drug synonyms“Did not appropriately control IVH; significantly increased rates of BTH and LDH excursions (LDH excursions is defined by LDH values > X2ULN) compared to ravulizumab.”
- terminated · 2024-10-01 · Alexion Pharmaceuticals, Inc. · Ravulizumab“Recruitment Challenges”
- terminated · 2024-09-25 · Alexion Pharmaceuticals, Inc. · Eculizumab“Pediatric clinical study is not feasible.”
- terminated · 2024-08-09 · Alnylam Pharmaceuticals · Cemdisiran“Study was terminated early having identified clinically meaningful magnitude of proteinuria reduction due to cemdisiran (study goal). Participants completed DBT period\&were in OLE period. Sponsor had no concerns with safety\&integrity of participants.”
- terminated · 2024-08-06 · Andrew Lowy · matched by the registry's drug synonyms“Insufficient funding”
- withdrawn · 2024-02-21 · Ophthotech Corporation · matched by the registry's drug synonyms“No patients were enrolled. Study terminated for portfolio reasons.”
- terminated · 2023-11-29 · Cedars-Sinai Medical Center · Eculizumab“PI moved institutions”
Paediatric incentives and obligations for C5's drugs
For vilobelimab, zilucoplan, avacincaptad pegol, crovalimab, pozelimab, eculizumab, ravulizumab, pexelizumab, nomacopan, cemdisiran, olendalizumab, tesidolumab: what EMA and FDA publish about paediatric plans, orphan status and pediatric approvals, and how many registered studies take children. Each line is a yes, no or not published fact from the record linked under it; none of it is a forecast.
- FDA pediatric target list: no · C5 is not on the listRule: Target is on FDA's Pediatric Molecular Target List (relevant targets). Source: FDA Pediatric Molecular Target List (Listed as February 2026 on the FDA Pediatric Oncology page; the file served is 9.7.22_the_relevant_molecular_target_list.xlsx, whose Summary of Changes sheet is headed 'SEPTEMBER 2022 UPDATE' (footnote date 2022-09-01)), 2026-11-11
- EU paediatric investigation plan: yes · EMA/PE/0000229605 (ravulizumab, Treatment in haematopoietic stem cell transplantation): PM: decision on the application for modification of an agreed PIP, 14 Apr 2025; EMA/PE/0000221604 (ravulizumab, Treatment of paroxysmal nocturnal haemoglobinuria): PM: decision on the application for modification of an agreed PIP, 3 Jan 2025; EMA/PE/0000221296 (ravulizumab, Treatment of atypical haemolytic uremic syndrome): PM: decision on the application for modification of an agreed PIP, 3 Jan 2025; EMA/PE/0000257243 (pozelimab, Treatment of myasthenia gravis): PM: decision on the application for modification of an agreed PIP, 4 Aug 2025; +18 moreRule: An EMA PIP decision (agreement, modification or waiver) lists the drug as its active substance, exact name. Source: EMA data download (dated snapshot), 2026-11-11
- EU orphan designation: yes · EU/3/22/2707 withdrawn 11 Oct 2022: Treatment of Guillain-Barré syndrome; EU/3/22/2650 withdrawn 18 Jul 2022: Treatment of myasthenia gravis; EU/3/21/2489 positive 20 Aug 2021: Treatment of primary IgA nephropathy; EU/3/23/2810 positive 25 Jul 2023: Treatment in haematopoietic stem cell transplantation; +13 moreRule: An EU orphan designation lists the drug as its active substance (exact name) or belongs to a medicine that does; withdrawn and expired designations are listed with their status. Source: EMA data download (dated snapshot), 2026-11-11
- Authorised in the EU: yes · Bekemv: authorised since 19 Apr 2023; Zilbrysq: authorised since 1 Dec 2023; Epysqli: authorised since 26 May 2023; Ultomiris: authorised since 2 Jul 2019; +4 moreRule: An EMA medicine record lists the drug as its active substance or INN, exact name; national-only authorisations are not in this file. Source: EMA data download (dated snapshot), 2026-11-11
- FDA orphan designation: not published · FDA's orphan designation database turns away automated requests, so this app does not read it; search it by hand at the linkRule: Would be: an FDA orphan designation names the drug. Source: FDA orphan designations search
- FDA pediatric oncology approval: no · FDA's table lists no pediatric approval of vilobelimab, zilucoplan, avacincaptad pegol, crovalimab, pozelimab, eculizumab, ravulizumab, pexelizumab, nomacopan, cemdisiran, olendalizumab, tesidolumabRule: FDA's Pediatric Oncology Drug Approvals table names the drug. Source: FDA Pediatric Oncology Drug Approvals (content current as of 2026-03-03), 2026-11-12
- Registered studies open to children: yes · 45 interventional studies of vilobelimab, zilucoplan, avacincaptad pegol, crovalimab, pozelimab, eculizumab, ravulizumab, pexelizumab, nomacopan, cemdisiran, olendalizumab, tesidolumab include patients under 18; 13 are recruiting, not yet recruiting or activeRule: The registry's age group includes Child (birth to 17 years), which also counts studies that set no minimum age. Source: ClinicalTrials.gov search, 2026-11-12
Rare pediatric disease voucher. FDA, content current as of 29 Apr 2026: “As of February 3, 2026, enactment of the Consolidated Appropriations Act, 2026, the rare pediatric disease PRV program will sunset after September 30, 2029. FDA may not award any PRVs under this program after September 30, 2029.” Voucher sale prices are not shown here; they appear only as company-reported news. FDA: Rare Pediatric Disease Designation and Priority Review Voucher Programs
All 13 drugs and clinical candidates as a table
Each drug, stage and indication links to its Open Targets record, where the trials and approvals behind it are listed. Salt forms (for example a mesylate) are listed as Open Targets records them, as separate entries.