Eculizumab is a monoclonal antibody indicated for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) or with atypical hemolytic uremic syndrome (aHUS). In Brazil in recent years eculizumab was the most expensive drug requested through court orders, obliging public health managers to import it from the USA. From 2012 to 2016, approximately BRL 424 million (USD 112 million) was spent on eculizumab. The purpose of this study was to assess the regulatory situation and the scientific evidence on the safety and efficacy of eculizumab.

A literature search was conducted in PubMed, The Cochrane Library, and the Centre for Reviews and Dissemination databases on September 2017. The websites of regulatory agencies were also searched.

In 2007, the use of eculizumab was approved by the United States Food and Drug Administration and the European Medicines Agency. In Brazil, despite the provision of eculizumab through judicial proceedings since 2009, the manufacturer of eculizumab only requested a licence for it in 2017, after several meetings with the government when the company agreed to provide the drug at approximately half the price of the imported product. The efficacy of eculizumab in PNH patients was assessed in one randomized, placebo controlled study, one single arm study, and one long-term extension study. The drug reduced hemolysis and the need for transfusion, although the studies had methodological problems. The efficacy of eculizumab in the treatment of aHUS was assessed in four prospective, controlled open-label studies, two long-term extension studies, and one retrospective study. Eculizumab normalized platelet counts and reduced the need for plasmapheresis, although the studies had no control group. Eculizumab was well tolerated, with no meningococcal infections occurring after patients were immunized.

Some companies have no interest in licensing their products in Brazil because their provision by judicial proceedings is more lucrative. This situation promotes litigation and irrational prescription of drugs, and also obligates the Brazilian government to import expensive health products.