Pegcetacoplan clinical trials
Pegcetacoplan is named as an intervention in 8 current clinical trials in our UK, USA, France, Spain, Czechia, Italy, Germany, Switzerland, Canada, Belgium, Australia, Netherlands, Brazil, South Korea, Mexico, Japan, Israel, Poland, Austria and Taiwan registers. 3 are recruiting patients now. The most common phase is Phase 2. The sponsor with the most trials is Apellis Pharmaceuticals, Inc (4). The most-studied condition is Geographic Atrophy. The EU clinical trials register (CTIS) separately lists 3 authorised or ongoing trials.
Where Pegcetacoplan is approved
All regulators →Of the three regulators tracked here, the first to approve Pegcetacoplan was the US, on 14 May 2021. It is approved in 3 of the 3 regulators tracked here.
- 1 EU orphan designation, most recently for treatment of C3 glomerulopathy with or without immune complexes (11 Nov 2022, positive)
From the regulators' own registers: FDA Drugs@FDA, the EMA medicines list and Health Canada's Notice of Compliance database. Dates are the first listed approval of a product containing only this substance; combination products and nationally authorised EU medicines are not counted.
Pegcetacoplan patents and exclusivity
Expiry tracker →Protections still running for marketed products containing Pegcetacoplan, as listed by the regulators. Expired patents and exclusivities, and discontinued products, are not shown.
- 15 listed patents; the last listed expires 14 Dec 2038 (US 12290566).
- Latest patent on the drug substance itself: 15 Nov 2033 (US 11292815).
- Exclusivity ODE-533: Orphan drug exclusivity: treatment of adult and pediatric patients aged 12 years and older with primary immune-complex membranoproliferativ…, until 28 Jul 2032.
- Exclusivity ODE-539: Orphan drug exclusivity: treatment of adult and pediatric patients aged 12 years and older with c3 glomerulopathy (c3g), to reduce proteinu…, until 28 Jul 2032.
- Exclusivity I-972: New indication: treatment of adult and pediatric patients aged 12 years and older with c3 glomerulopathy (c3g) or primary immune-complex me…, until 28 Jul 2028.
- Exclusivity ODE-351: Orphan drug exclusivity: treatment of adult patients with paroxysmal nocturnal hemoglobinuria (pnh), until 14 May 2028.
- ASPAVELI for treatment of paroxysmal nocturnal haemoglobinuria: until 14 Dec 2031. Union Register
- ASPAVELI for treatment of C3 glomerulopathy with or without immune complexes: until 16 Jan 2036. Union Register
Reproduced from the FDA Orange Book and Purple Book (checked 8 Nov 2026) and the European Commission's Union Register of medicinal products (CC BY 4.0; the EU expiry date is calculated exactly as the Commission's register displays it). Listed patents and dates are as submitted to or published by the regulator and may not reflect litigation, invalidation, settlements, lapsed fees or later extensions. This is not legal advice or a freedom-to-operate opinion, and it does not say when a generic or biosimilar will launch.
What health systems spend on Pegcetacoplan
All medicines →From NHSBSA Prescription Cost Analysis (Open Government Licence v3.0) and CMS State Drug Utilization Data. Both are gross amounts: neither system publishes its discounts or rebates per drug. Products combining several substances are not counted here.
How Pegcetacoplan works
All drug targets →- Complement C3 inhibitor InhibitorTarget: C3 complement C3ChEMBL only
- Age-related macular degenerationPhase 3
- Atrophic macular degenerationPhase 3
- Cold agglutinin diseasePhase 3
- Focal segmental glomerulosclerosisPhase 2/3
- IgA glomerulonephritisPhase 2
- Amyotrophic lateral sclerosisPhase 2
- Autoimmune hemolytic anemiaPhase 2
- Fallopian tube cancerPhase 2
- Immunoglobulin-mediated membranoproliferative glomerulonephritisPhase 2
- Lupus nephritisPhase 2
Mechanisms and diseases from ChEMBL via the Open Targets Platform (release 26.09), ChEMBL record CHEMBL4298211, CC BY-SA 3.0. Targets checked against the IUPHAR/BPS Guide to PHARMACOLOGY (2026.3.1), ligand 13826, CC BY-SA 4.0. Trial phases are not approvals: approved uses are shown from the regulators' own records where we hold them.
NICE appraisals of Pegcetacoplan
All NICE appraisals →NICE, which decides whether the NHS in England should fund new medicines, has published 1 technology appraisal of Pegcetacoplan. 1 is current, and 1 of it recommends it for at least some patients.
| Appraisal | NICE recommendation | Published | Status |
|---|---|---|---|
| Pegcetacoplan for treating paroxysmal nocturnal haemoglobinuria NICE TA778 | Recommended | 9 Mar 2022 | Current |
NICE decisions apply to the NHS in England. In Ireland the HSE decides on reimbursement, advised by the National Centre for Pharmacoeconomics. Titles, categories and dates link to the guidance on nice.org.uk. © NICE 2026 technology appraisal guidance. Available from www.nice.org.uk/guidance. All rights reserved. Subject to Notice of rights. NICE guidance is prepared for the National Health Service in England. All NICE guidance is subject to regular review and may be updated or withdrawn. NICE accepts no responsibility for the use of its content in this product/publication.
Patient leaflets for Pegcetacoplan
The package leaflet is the official guide for patients that comes with every medicine. These links go to the regulators that publish it.
- IEIreland: 1 product on the HPRA listEach product record links to the HPRA, which publishes the leaflet and SmPC.
- EUEuropean Union: AspaveliEMA product information: package leaflet and summary of product characteristics in every EU language.
- UKUnited Kingdom: Search MHRA ProductsPatient information leaflets and SmPCs for UK-licensed products.
- USUnited States: Search DailyMedFDA-approved labels, including patient and medication guides.
Leaflets differ between brands and countries; always read the one that comes with your own medicine. This is regulatory information, not medical advice.
Reference identifiers for Pegcetacoplan
- ChEMBL
- CHEMBL4298211
- DrugBank
- DB16694
- PubChem CID
- 405226480
- CAS number
- 2019171-69-6
- FDA UNII
- TO3JYR3BOU
- Wikidata
- Q106863504
- Wikipedia
- Pegcetacoplan
Codes that identify this medicine in other databases, from Wikidata (CC0), matched by its ChEMBL ID. The ATC code is the WHO classification of what the medicine is used for.
Trials by status
Trials by phase
Clinical trials of Pegcetacoplan
8 trialsRegisters list studies that are recruiting, about to open, active but closed to enrolment, or enrolling by invitation. The UK and USA registers cover industry-sponsored studies only. Trial titles and statuses are copied from the registry as published.
Pegcetacoplan in the EU trials register (CTIS)
3 trialsAuthorised or ongoing trials in the EU Clinical Trials Information System that list a product containing Pegcetacoplan. Many trials are registered in both CTIS and ClinicalTrials.gov, so these are shown separately rather than added to the count above. Trials with a site in Ireland are listed first.
Pegcetacoplan across our data
Ireland · EU · USThe same active substance in our other registers, matched by its exact name. Each entry links to the register record it comes from.
Licensed in Ireland (HPRA)
1 single-substance product containing Pegcetacoplan on the HPRA medicines list.
- ASPAVELI 1 080 mg solution for infusionApellis Ireland Limited · EU/1/21/1595/001-002
Supply shortages
No entry for Pegcetacoplan in the EMA, FDA or German BfArM shortage lists we track.
Conditions studied with Pegcetacoplan
14 conditionsAll 14 conditions
| Condition (as registered) | Trials |
|---|---|
| Geographic Atrophy | 2 |
| Paroxysmal Nocturnal Hemoglobinuria (PNH) | 1 |
| Paroxysmal Hemoglobinuria | 1 |
| C3 Glomerulopathy (C3G), Primary Immune-Complex Membranoproliferative Glomerulonephritis (IC-MPGN) | 1 |
| Recurrent Glomerular Disease After Kidney Transplantation | 1 |
| C3G | 1 |
| IC-MPGN | 1 |
| C3 Glomerulopathy | 1 |
| C3 Glomerulonephritis | 1 |
| Paroxysmal Nocturnal Hemoglobinuria | 1 |
| C3 Glomerulopathy (C3G) | 1 |
| Immune Complex Membranoproliferative Glomerulonephritis (IC-MPGN) | 1 |
| Macular Degeneration, Age-related | 1 |
| Geographic Atrophy Secondary to Age-related Macular Degeneration | 1 |
Sponsors
lead sponsor per trial| Lead sponsor | Trials |
|---|---|
| Apellis Pharmaceuticals, Inc | 4 |
| Swedish Orphan Biovitrum | 3 |
| Boehringer Ingelheim | 1 |
By country register
A trial with sites in more than one country appears in each of those registers. CTIS does not publish a recruiting flag; its trials may also appear in the registers above.
Related compounds
Questions about Pegcetacoplan trials
How many clinical trials are testing Pegcetacoplan?
8 current trials in our UK, USA, France, Spain, Czechia, Italy, Germany, Switzerland, Canada, Belgium, Australia, Netherlands, Brazil, South Korea, Mexico, Japan, Israel, Poland, Austria and Taiwan registers name Pegcetacoplan (also registered as APL-2) as a drug or biological intervention, as of the registry sync on 9 November 2026. Completed and withdrawn studies are not counted.
Are Pegcetacoplan trials recruiting patients?
Yes. 3 of the 8 trials are recruiting: 2 with sites in UK, 2 with sites in USA, 2 with sites in France, 2 with sites in Spain, 2 with sites in Czechia, 1 with sites in Italy, 1 with sites in Germany, 1 with sites in Switzerland, 1 with sites in Canada, 1 with sites in Belgium, 1 with sites in Australia, 1 with sites in Netherlands, 1 with sites in South Korea, 1 with sites in Mexico, 1 with sites in Austria and 1 with sites in Taiwan. Eligibility and site contacts are on each trial's record. Only the study team can confirm whether a patient is eligible.
Who sponsors Pegcetacoplan clinical trials?
Apellis Pharmaceuticals, Inc (4), Swedish Orphan Biovitrum (3) and Boehringer Ingelheim (1). The number in brackets is how many trials each organisation leads.
Is Pegcetacoplan licensed in Ireland?
Yes. The HPRA medicines list includes 1 single-substance product containing Pegcetacoplan, for example ASPAVELI 1 080 mg solution for infusion (Apellis Ireland Limited). A licence does not mean a product is currently marketed or reimbursed; see each product's record.
Is there a Pegcetacoplan shortage?
Not in the lists we track: Pegcetacoplan has no entry in the EMA, FDA or German BfArM shortage registers as of their latest sync.
Where does this information come from?
Every trial, status, phase and sponsor on this page is copied from public ClinicalTrials.gov records by a daily sync. Nothing here describes how Pegcetacoplan works or whether it is effective, and it is not medical advice.
How this page is built
Everything on this page is copied from public ClinicalTrials.gov registry records — it is not written from memory, and nothing here describes how Pegcetacoplan works or whether it is effective. Each trial links to its own record so you can check it yourself.
- ClinicalTrials.gov (U.S. National Library of Medicine) ↗ Trial IDs, titles, intervention names and synonyms, phase, overall status, lead sponsor and conditions for every trial on this page
This page is built automatically: a daily sync job reads the registry records behind our country trial registers, collects every trial that lists Pegcetacoplan (or a registered synonym) as a drug or biological intervention, and tabulates the registry fields directly. Placebos and generic comparators are excluded. There is no AI drafting and no per-compound editorial pass, and the page has not been clinically reviewed. The “last verified” date above is the timestamp of that sync, not a review date. Our general editorial approach is described in our editorial standards and corrections policy. The ClinicalTrials.gov data on this page was last retrieved on 9 Nov 2026. How every register is built: methodology · fixes we have made: corrections log.