Pegcetacoplan is under clinical development by Apellis Pharmaceuticals and currently in the Phase II, Phase III and Pre-Registration in clinical pathway. The characteristics of the clinical trial as well as other attributes related to the drug, regulations, and company play a fundamental role in ensuring the likelihood of transition that the drug moves from its current development stage to next.

According to GlobalData, the latest event to affect Pegcetacoplan’s likelihood of approval (LoA) and phase transition for Glomerulonephritis took place on 03 Oct 2022, which increased the likelihood that the drug progresses to the next phase in its clinical pathway and increased the likelihood of final approval for this indication.

GlobalData uses proprietary data and analytics to provide a complete picture of this assessment in their Pegcetacoplan Likelihood of Approval (LoA) and Phase Transition Success Rate (PTSR) Report.

Pegcetacoplan overview

Pegcetacoplan (Empaveli, Aspaveli) is a synthetic peptide acts as a complement inhibitor. It is formulated as injectable solution for subcutaneous route of administration. Empaveli is indicated for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH), Aspaveli is indicated in the treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH) who are anaemic after treatment with a C5 inhibitor for at least 3 months.

Pegcetacoplan (APL-2) is under development for the treatment of post-transplant recurrence of C3G or IC-MPGN, transplant-associated thrombotic microangiopathy (TA-TMA), IgA nephropathy (Berger's disease), lupus nephritis, primary membranous nephropathy, or C3 glomerulopathy (Kidney Disease (Nephropathy)) includes C3 glomerulonephritis, paroxysmal nocturnal hemoglobinuria (PNH), geographic atrophy (advanced form of dry age-related macular degeneration) and autoimmune hemolytic anemia or cold agglutinin disease(wAIHA) and amyotrophic lateral sclerosis. It is a  synthetic cyclic peptide conjugated to a polyethylene glycol (PEG) polymer. It is administered subcutaneously as an infusion for PNH and intravitreally for AMD. It is a compstatin derivative and a long-acting small cyclic peptide that targets complement factor C3. It was also under development for the treatment of kidney transplant rejection, refractory myasthenia gravis and neuromyelitis optica, neovascular age-related macular degeneration (AMD).

Apellis Pharmaceuticals overview

Apellis Pharmaceuticals (Apellis), is a clinical-stage biopharmaceutical company that focuses on the discovery and development of therapeutic compounds for autoimmune and inflammatory diseases through inhibition of the complement system at the level of C3 (central protein). Its pipeline product portfolio includes APL-2, a C3 inhibitor administered by intravitreal injections and subcutaneous injections for the treatment of geographic atrophy (GA), paroxysmal nocturnal hemoglobinuria (PNH), wet age-related macular degeneration (AMD), autoimmune hemolytic anemia (AIHA) and complement-dependent nephropathies. Apellis complement system activates C3 protein which enables immune responses such as opsonization, inflammation and formation of the membrane attack complex. It also conducts research on chronic therapy with a C3 inhibitor into clinical trials. The company operates with offices in the US, Ireland, Switzerland and Australia. Apellis is headquartered in Waltham, Massachusetts, the US.

Quick View Pegcetacoplan LOA Data

Report Segments
  • Innovator (NME)
Drug Name
  • Pegcetacoplan
Administration Pathway
  • Intravitreal
  • Subcutaneous
Therapeutic Areas
  • Cardiovascular
  • Central Nervous System
  • Genito Urinary System And Sex Hormones
  • Hematological Disorders
  • Immunology
  • Ophthalmology
Key Developers
Highest Development Stage
  • Marketed


GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.

GlobalData’s Likelihood of Approval analytics tool dynamically assesses and predicts how likely a drug will move to the next stage in its clinical pathway (PTSR), as well as how likely the drug will be approved (LoA). This is based on a proprietary algorithm built from the drugs’ sales forecast, regulatory milestones, cost forecasts, WACC rate and other proprietary data sources found on GlobalData’s Pharmaceutical Intelligence Center.