Ravulizumab LA is under clinical development by Alexion Pharmaceuticals and currently in Phase II for IgA Nephropathy (Berger’s Disease). According to GlobalData, Phase II drugs for IgA Nephropathy (Berger’s Disease) have a 53% phase transition success rate (PTSR) indication benchmark for progressing into Phase III. GlobalData’s report assesses how Ravulizumab LA’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.

Ravulizumab LA overview

Ravulizumab (Ultomiris) is a humanized monoclonal antibody. It is formulated as solution and concentrate solution (long acting) for intravenous route of administration. Ultromiris is indicated for the treatment of paroxysmal nocturnal hemoglobinuria (PNH), in adults and pediatric patients one month of age and older. Ultomiris is also indicated for the treatment of adults and pediatric patients with atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy. Ultomiris is indicated for the treatment of adult patients with generalised myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody-positive and whose symptoms are difficult to control with high-dose intravenous immunoglobulin therapy (IVIg) or plasmapheresis.

(ALXN-1210, ultomiris) is under development for the treatment of generalized myasthenia gravis (gMG), primary progressive multiple sclerosis, glomerulonephritis-IgA nephropathy (Berger's disease), cardiac surgery-associated acute kidney injury, atypical hemolytic uremic syndrome (aHUS) pediatric and adults,neuromyelitis optica spectrum disorder (NMOSD), amyotrophic lateral sclerosis, dermatomyositis, thrombotic microangiopathy. It is a next-generation Soliris (eculizumab). It is administered intravenously and subcutaneously. The drug candidate is a long-acting anti-C5 antibody act by targeting complement C5 protein. It is developed based on Xencor's Xmab technology that utilizes Xtend Fc domain to enhance the half-life.

It was under development for treatment of severe pneumonia and acute lung injury or acute respiratory distress syndrome (ARDS) with COVID-19

Alexion Pharmaceuticals overview

Alexion Pharmaceuticals (Alexion) discovers, develops, and markets therapies based on complement biology and inhibition for rare diseases. Deep understanding of rare disease enables the company to innovate and enter new areas, where there is a great unmet medical need. The company’s portfolio spans complement inhibitors for the treatment of patients with anti-acetylcholine receptor (AChR) antibody-positive generalized myasthenia gravis (gMG), atypical hemolytic uremic syndrome (aHUS), paroxysmal nocturnal hemoglobinuria (PNH), and neuromyelitis optica spectrum disorder (NMOSD) in anti-aquaporin-4 (AQP4) antibody positive patients. The company also offers novel enzyme replacement therapies for patients with lysosomal acid lipase deficiency (LAL-D), hypophosphatasia (HPP), and ultra-rare metabolic disorders. Alexion is headquartered in New Haven, Connecticut, the US.

For a complete picture of Ravulizumab LA’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.