Ravulizumab

Ravulizumab is a potent and selective complement 5 (C5) inhibitor. It is a humanized monoclonal IgG2/4 kappa antibody produced in Chinese hamster ovary (CHO) cells. Ravulizumab was engineered from eculizumab, another complement inhibitor, to increase the duration of action and reduce the frequency of drug administration. It works by blocking terminal complement-mediated inflammation, cell activation, and cell lysis in blood disorders associated with the destruction of red blood cells, thrombosis, and impaired bone marrow function.

Ravulizumab was first approved by the FDA on December 21, 2018, for the treatment of paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome in children and adults. It was later approved by the European Commission on July 2, 2019, for the same indications. Ravulizumab is also used to treat myasthenia gravis. Ravulizumab is currently being investigated for the treatment of Coronavirus disease (COVID-19)-induced microvasculature injury and endothelial damage leading to thrombotic microangiopathy (TMA) causing acute kidney injury (AKI).

Ravulizumab is indicated for the treatment of adult and pediatric patients one month of age and older with paroxysmal nocturnal hemoglobinuria (PNH).

It is also indicated for the treatment of adult and pediatric patients one month of age and older with atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy (TMA). However, the FDA advises against the use of ravulizumab for the treatment of patients with Shiga toxin E. coli related hemolytic uremic syndrome (STEC-HUS).

Ravulizumab is also indicated for treatment of adult patients with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody-positive.

The European Commission approved ravulizumab for the treatment of paroxysmal nocturnal haemoglobinuria (PNH) in adults and children with a body weight of 10 kg or more with the following conditions: hemolysis with clinical symptoms indicative of high disease activity or clinically stable after having been treated with eculizumab for at least the past six months. Ravulizumab is also indicated for the treatment of hemolytic uremic syndrome (aHUS) in patients with a body weight of 10 kg or more who are either complement inhibitor treatment-naïve or have received eculizumab for at least 3 months and have evidence of response to eculizumab.