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  • New Oral Treatment Enhances Management of a Rare Blood Disorder

London, UK — AstraZeneca declared on Monday a significant milestone in the treatment of paroxysmal nocturnal haemoglobinuria (PNH), a rare and life-threatening blood disorder, with the approval of Voydeya (danicopan) in the United States.

The oral administration of Voydeya also marks a departure from the intravenous delivery methods of existing treatments, potentially offering greater convenience and flexibility for patients. This new therapy, developed as an oral, first-in-class Factor D inhibitor, is now approved as an add-on to existing treatments, Ultomiris (ravulizumab) or Soliris (eculizumab). Approximately 10-20% of individuals with PNH continue to experience severe EVH, leading to a substantial impact on their quality of life and health.

Addressing the Unmet Needs in PNH

PNH has historically posed treatment challenges, particularly in managing extravascular haemolysis (EVH), where red blood cells are destroyed outside the bloodstream. Until now, standard treatments like Ultomiris and Soliris, known as C5 inhibitors, have been unable to fully alleviate EVH symptoms in a significant portion of patients.

The Role and Impact of Voydeya

Voydeya’s approval heralds a new era in personalized treatment for PNH. By inhibiting Factor D, a key protein in the complement system involved in the development of PNH, Voydeya works in tandem with C5 inhibitors to provide a more comprehensive approach to managing this complex condition.


The approval of Voydeya is a testament to the progress in understanding and treating complex conditions like PNH. For patients who have struggled with managing EVH, this offers a promising new avenue for relief and improved health outcomes. As AstraZeneca continues to pioneer in the field of rare diseases, the PNH community looks forward to the positive impacts this new therapy will bring.

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By Lisa Luckas

Lisa Luckas is a Sr. Business News Editor at Nobot.News.

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