Pegcetacoplan
Complement Inhibitor — ANTINEOPLASTIC AND IMMUNOMODULATING AGENTS
Also known as: APL-2, aspaveli, empaveli
Data last refreshed: (DrugCentral release 2024-11-01)
Key facts
Pegcetacoplan is a drug in the Complement Inhibitor class catalogued in DrugCentral. It was first approved by the FDA in 2021. Its mechanism of action is classified as Complement Inhibitors. DrugCentral lists 1 molecular target, including C3. Recorded indications include Geographic atrophy secondary to age-related macular degeneration and Paroxysmal nocturnal hemoglobinuria.
- Drug class: Complement Inhibitor.
- Mechanism of action: Complement Inhibitors.
- Molecular targets: C3 (1 listed in DrugCentral).
- Indications: Geographic atrophy secondary to age-related macular degeneration and Paroxysmal nocturnal hemoglobinuria.
- ATC code: L04AJ03 (Complement inhibitors).
Indications & contraindications
Drug–disease relationships curated by DrugCentral (OMOP / SNOMED concepts).
Indications (2)
- Geographic atrophy secondary to age-related macular degeneration
- Paroxysmal nocturnal hemoglobinuria
Contraindications (4)
- Ocular or periocular infection
- Unresolved serious infection caused by Haemophilus influenzae
- Unresolved serious infection caused by Neisseria meningitidis
- Uveitis
Mechanism & targets
| Mechanism of action (MoA) | Complement Inhibitors |
|---|---|
| Established pharmacologic class | Complement Inhibitor |
Molecular targets (1)
| Target | Gene | Action | Activity |
|---|---|---|---|
| Complement C3 | C3 | inhibitor | IC50 6.60 |
Regulatory approvals (3)
| Date | Agency | Marketed as | Applicant | Orphan |
|---|---|---|---|---|
| 2023-03-27 | PMDA | — | Swedish Orphan Biovitrum Japan Co., Ltd. | |
| 2021-12-13 | EMA | — | SWEDISH ORPHAN BIOVITRUM AB (PUBL) | Yes |
| 2021-05-14 | FDA | — | APELLIS PHARMACEUTICALS INC | Yes |
ATC classification
| ATC code | Therapeutic hierarchy |
|---|---|
L04AJ03 | ANTINEOPLASTIC AND IMMUNOMODULATING AGENTS › IMMUNOSUPPRESSANTS › IMMUNOSUPPRESSANTS › Complement inhibitors |
S01XA31 | SENSORY ORGANS › OPHTHALMOLOGICALS › OTHER OPHTHALMOLOGICALS › Other ophthalmologicals |
Reported adverse-event signals
Strongest FDA FAERS disproportionality signals (log-likelihood ratio). A signal is a statistical association in spontaneous reports, not proof that the drug causes the event.
| MedDRA term | LLR |
|---|---|
| Breakthrough haemolysis | 526.9 |
| Choroidal neovascularisation | 418.2 |
| Vitritis | 401.5 |
| Vitreous floaters | 397.6 |
| Neovascular age-related macular degeneration | 335.2 |
| Haemolysis | 286.4 |
| Uveitis | 274.3 |
| Eye complication associated with device | 234.4 |
| Eye inflammation | 229.6 |
| Retinal vasculitis | 226.4 |
| Visual impairment | 223.8 |
| Retinal haemorrhage | 215.5 |
Chemical structure
| Molecular formula | — |
|---|---|
| Molecular weight | — |
| CAS number | 2019171-69-6 |
| InChIKey | — |
| PubChem CID | — |
Frequently asked questions
Answers are generated from the structured data on this page, not written by hand.
What is Pegcetacoplan used for?
DrugCentral lists the following indications for Pegcetacoplan: Geographic atrophy secondary to age-related macular degeneration and Paroxysmal nocturnal hemoglobinuria.
How does Pegcetacoplan work?
Pegcetacoplan is classified as Complement Inhibitors; its molecular targets in DrugCentral include C3 (inhibitor).
When was Pegcetacoplan first approved?
Pegcetacoplan was first approved by the FDA in 2021.
What drug class does Pegcetacoplan belong to?
Pegcetacoplan is in the Complement Inhibitor class.
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Sources
- DrugCentral drug card 5457 — structure, indications, targets, approvals, pharmacologic classes (CC BY-SA 4.0)
- ChEMBL CHEMBL4298211 — compound record and bioactivity
- DrugBank DB16694 — drug record
- FDA UNII TO3JYR3BOU — substance identity
- DailyMed: FDA labels for Pegcetacoplan — current prescribing information (label search)
- UniProt P01024 (C3) — target protein
- FDA Adverse Event Reporting System (openFDA FAERS) — adverse-event signals, as aggregated by DrugCentral
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