Drugs / Pegcetacoplan
Pegcetacoplan
Regulatory milestones approvals, filings & regulatory actions · 6 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Label expansions (2) | US (FDA) | EMPAVELI | — | 2023-02-08 – 2025-07-28 | fda.gov ↗ |
| Filed | EU (EMA) | Aspaveli | C3 glomerulopathy, primary IC-MPGN | 2025-02-20 | “the European Medicines Agency (EMA) has validated an indication extension application for Aspaveli® (pegcetacoplan) for the treatment of C3 glomerulopathy (C3G) and primary...” sobi.com ↗ |
| Refused (EMA) | EU (EMA) | Syfovre | — | 2024-12-16 | europa.eu ↗ |
| Approved | EU (EMA) | Aspaveli | — | 2021-12-13 | europa.eu ↗ |
| Approved | US (FDA) | EMPAVELI | — | 2021-05-14 | fda.gov ↗ |
Trials 30
News releases announcing trial results or a regulatory action · 17
| Date | Issuer | Release |
|---|---|---|
| 2026-01-16 | Sobi, Inc. | Regulatory Sobi Receives European Commission Approval for Aspaveli® (pegcetacoplan) for the Treatment of C3G and Primary IC-MPGN sobi.com ↗
Aspaveli is now available for patients 12 years and older with these serious conditions. |
| 2025-12-12 | Sobi, Inc. | Regulatory Sobi Receives Positive CHMP Opinion for Aspaveli® (pegcetacoplan) for the Treatment of C3G and Primary IC-MPGN sobi.com ↗ |
| 2025-12-04 | Sobi, Inc. | Results The New England Journal of Medicine Publishes Positive Phase 3 VALIANT Results of pegcetacoplan for C3G and Primary IC-MPGN sobi.com ↗
The study met its primary endpoint, demonstrating a statistically significant 68% (p < 0.0001) proteinuria reduction in pegcetacoplan-treated patients compared to placebo. |
| 2025-11-05 | Sobi, Inc. | Results Sobi to present New One-Year Data on pegcetacoplan's efficacy in C3G and primary IC-MPGN and new outcome data on NASP at ASN Kidney Week sobi.com ↗
New one-year data reinforces the robust and sustained efficacy of Aspaveli/Empaveli (pegcetacoplan) in patients with C3G and primary IC-MPGN. |
| 2025-06-06 | Sobi, Inc. | Results Sobi and Apellis: Aspaveli®/Empaveli® Demonstrates Sustained One-Year Efficacy in Phase 3 Study for Rare Kidney Diseases sobi.com ↗
In the VALIANT study, Aspaveli demonstrated a statistically significant 68% proteinuria reduction versus placebo at Week 26, which was sustained at one year. |
| 2025-02-20 | Sobi, Inc. | Regulatory EMA validates indication extension application for Aspaveli® for treatment of C3G and primary IC-MPGN sobi.com ↗
The EMA validation leads us one step closer to potentially bringing this treatment to European patients |
| 2024-10-26 | Sobi, Inc. | Results Pivotal VALIANT results presented at Kidney Week show pegcetacoplan treatment effect in patients with C3G / primary IC-MPGN sobi.com ↗ |
| 2024-08-08 | Sobi, Inc. | Results Sobi and Apellis announce positive topline results from phase 3 VALIANT study of pegcetacoplan in C3G and primary IC-MPGN sobi.com ↗
Met the primary endpoint, achieving statistically significant 68% (p < 0.0001) reduction in proteinuria compared to placebo in a broad study population |
| 2024-05-24 | Sobi, Inc. | Results Positive data from phase 2 NOBLE study of pegcetacoplan presented as late-breaker at ERA congress sobi.com ↗
Sobi® and Apellis Pharmaceuticals, Inc. today announced positive one-year results from the phase 2 NOBLE study investigating systemic pegcetacoplan, a targeted C3 therapy, for the treatment of post-transplant recurrence of C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN). |
| 2024-05-08 | Sobi, Inc. | Regulatory Aspaveli® (pegcetacoplan) approved in Europe for use among treatment naïve adult patients with PNH sobi.com ↗
Sobi® today announced that the European Commission (EC) has approved an indication extension for Aspaveli® (pegcetacoplan) for treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH) who have haemolytic anaemia. |
| 2023-10-17 | Sobi, Inc. | Results Sobi announces positive Phase 2 NOBLE results of pegcetacoplan in post-transplant recurrence of primary IC-MPGN and C3G sobi.com ↗
Five (50%) patients showed a reduction in C3c staining by two or more orders of magnitude of intensity from baseline. |
| 2022-06-10 | Sobi, Inc. | Results New data reinforce robust efficacy and safety profile of Aspaveli/Empaveli (pegcetacoplan) for PNH at the EHA2022 Congress sobi.com ↗
Patients treated with Aspaveli/Empaveli showed significant improvements compared to C5 inhibitors across all key disease measures evaluated, including lactate dehydrogenase normalisation, haemoglobin stabilisation and transfusion avoidance at week 26. |
| 2021-12-13 | Sobi, Inc. | Results Aspaveli®/Empaveli™ (pegcetacoplan) demonstrated sustained normalisation of clinical measures in a broad PNH patient population sobi.com ↗ |
| 2021-10-15 | Sobi, Inc. | Regulatory Aspaveli® (pegcetacoplan) receives positive CHMP opinion for treatment of PNH sobi.com ↗
The positive opinion from the CHMP is now referred to the European Commission for an approval decision. |
| 2021-05-25 | Sobi, Inc. | Results Positive top-line results from the phase 3 PRINCE study of pegcetacoplan in treatment-naïve patients with PNH sobi.com ↗
Pegcetacoplan demonstrated statistical superiority on the co-primary endpoints of haemoglobin stabilization and reduction in lactate dehydrogenase (LDH) compared to standard of care, which did not include complement inhibitors, at week 26. |
| 2021-03-17 | Sobi, Inc. | Results The NEJM publishes phase 3 PEGASUS study results comparing pegcetacoplan to eculizumab for PNH sobi.com ↗ |
| 2020-12-10 | Sobi, Inc. | Results Sobi and Apellis report positive top-line results at 48 weeks from the phase 3 PEGASUS study of pegcetacoplan in PNH sobi.com ↗
Treatment with pegcetacoplan resulted in a sustained improvement in haemoglobin with a mean increase from baseline of 2.7 g/dL at week 48, which is equal to the 2.7 g/dL increase seen at week 16 with pegcetacoplan-treated patients |
All press releases naming this drug 23 releases
Evidence & citations 9 cited values
Every value below carries the sentence it was read from. 37 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Pegcetacoplan | ClinicalTrials.gov intervention name — accepted as the source's own label NCT04572854 ↗ |
| Known as | APL-2 | “A Phase 2, Randomized, Placebo Controlled, Multicenter, Masked Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacodynamics of Multidose APL 3007 in Combination...” NCT07215390 ↗9“PEGCETACOPLAN (APL-2)” NCT03777332 ↗ “Pegcetacoplan (APL-2)” NCT04579666 ↗ “APL-2, Pegcetacoplan” NCT07214740 ↗ |
| Known as | Aspaveli | ChEMBL registry synonym — accepted as the source's own label CHEMBL4298211 ↗ |
| Known as | Empaveli | ChEMBL registry synonym — accepted as the source's own label CHEMBL4298211 ↗ |
| Known as | Syfovre | “A Phase 2, Randomized, Placebo Controlled, Multicenter, Masked Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacodynamics of Multidose APL 3007 in Combination...” NCT07215390 ↗ |
| Action | Inhibit | “Complement (C3) Inhibitor” NCT02264639 ↗ |
| Modality | Peptide | “Pegcetacoplan, a pegylated peptide targeting proximal complement protein C3” PMID 33730455 ↗ Mar 2021 |
| Route | Intravitreal | “will be administered via intravitreal injection” NCT02503332 ↗ |
| Target | C3 | “inhibits C3” PMID 35869170 ↗ Jul 2022 |