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<p>The development of several complement-inhibiting medicines has been a major advance in the management of paroxysmal nocturnal hemoglobinuria (PNH), a rare and life-threatening disease. Patients now have a life expectancy close to that of the general population, and the quality of life of most patients has improved substantially. In addition, by targeting either the proximal or the terminal complement pathway, we have also been able to improve our understanding of the pathophysiology of PNH. Within the landscape of this rather spectacular progress, a major drawback has been the rather astronomic price of all of these medicines: This means, in practice, that in many countries they are not available at all, and a conservative estimate is that at least one-half of the PNH patients in the world have no access to complement inhibitors. It is urgent to correct this inequality that is also an injustice. Very recently, the Max Foundation, a global nonprofit organization, has announced that it will collaborate with a pharmaceutical company to make ‘innovative treatment for PNH’ available in many countries: We may now hope that other companies will join such collaborations.</p>

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Complement inhibitor therapy should be available to patients with paroxysmal nocturnal hemoglobinuria throughout the world

  • 期刊论文

  • 发表日期：

    2026年8月4日

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  • Lucio Luzzatto

摘要

The development of several complement-inhibiting medicines has been a major advance in the management of paroxysmal nocturnal hemoglobinuria (PNH), a rare and life-threatening disease. Patients now have a life expectancy close to that of the general population, and the quality of life of most patients has improved substantially. In addition, by targeting either the proximal or the terminal complement pathway, we have also been able to improve our understanding of the pathophysiology of PNH. Within the landscape of this rather spectacular progress, a major drawback has been the rather astronomic price of all of these medicines: This means, in practice, that in many countries they are not available at all, and a conservative estimate is that at least one-half of the PNH patients in the world have no access to complement inhibitors. It is urgent to correct this inequality that is also an injustice. Very recently, the Max Foundation, a global nonprofit organization, has announced that it will collaborate with a pharmaceutical company to make ‘innovative treatment for PNH’ available in many countries: We may now hope that other companies will join such collaborations.