Emerging treatments
Crovalimab
An anti-C5 recycling monoclonal antibody administered subcutaneously. In one phase 3 noninferiority study, 79.3% of participants receiving crovalimab achieved hemolysis control from week 5 to week 25 compared with 79.0% of individuals treated with eculizumab.[59] Crovalimab was noninferior to eculizumab in the co-primary endpoint of transfusion avoidance (65.7% vs. 68.1%, respectively).[59] The safety profiles of crovalimab and eculizumab were similar.[59] Crovalimab is approved by the Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the treatment of patients with PNH.
Danicopan
Danicopan is an oral factor D inhibitor intended to be used as an add-on to C5 inhibitor therapy for patients with PNH experiencing clinically significant extravascular hemolysis (EVH). In one small phase 3 randomized double-blind placebo-controlled trial, danicopan combined with ravulizumab or eculizumab significantly improved fatigue and anemia, and reduced transfusion dependence, at 12 weeks.[60] Danicopan is approved by the FDA as add-on therapy to ravulizumab or eculizumab for the treatment of extravascular hemolysis in adults with PNH. In Europe, danicopan is indicated as an add-on to ravulizumab or eculizumab for the treatment of adult patients with PNH who have residual hemolytic anemia.
Gene therapy
Although the gene whose mutations are responsible for PNH is known and sequenced, gene therapy (the replacement of the mutant gene with a normal gene) is unlikely to work, as the influences that caused the defective clone to expand would presumably still be operative. A normal clone persists in most cases of PNH.
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