Zilucoplan is under clinical development by Ra Pharmaceuticals and currently in Pre-Registration for Myasthenia Gravis. According to GlobalData, Pre-Registration drugs for Myasthenia Gravis have a 100% phase transition success rate (PTSR) indication benchmark for progressing into Marketed. GlobalData’s report assesses how Zilucoplan’s drug-specific PTSR and Likelihood of Approval (LoA) scores compare to the indication benchmarks. Buy the report here.

GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data. Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval.

Zilucoplan overview

Zilucoplan is under development for the treatment of Coronavirus disease 2019 (COVID-19) patients with acute hypoxic respiratory failure, atypical hemolytic uremic syndrome, lupus nephritis, amyotrophic lateral sclerosis, renal disorder  and generalized myasthenia gravis. The drug candidate is administered through the subcutaneous route. The drug candidate is synthetic 15-amino-acid macrocyclic peptide acylated with a polyethyleneglycol palmitoylated like. It acts by targeting complement C5. It was also under development for paroxysmal nocturnal hemoglobinuria (PNH).

It was under development for the treatment of immune-mediated necrotizing myopathy (Autoimmune-musculoskeletal disorder).

Ra Pharmaceuticals overview

Ra Pharmaceuticals, a subsidiary of UCB SA is a clinical stage biopharmaceutical company that develops therapeutics for patients with rare diseases based on its Extreme Diversity technology platform. It has pipeline products in pre-clinical, Phase I and Phase II trials for CNS diseases, autoimmune and renal disorders. The company’s lead product candidate, Zilucoplan, is a self-administered subcutaneous (SC) injection for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) and generalized myasthenia gravis (gMG). The company has collaboration with other companies to carry out the pre-clinical programs for glomerulonephritis (C3GN), and dense deposit disease (DDD). Ra Pharmaceuticals is headquartered in Cambridge, Massachusetts, the US.

For a complete picture of Zilucoplan’s drug-specific PTSR and LoA scores, buy the report here.


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.