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Paroxysmal Nocturnal Hemoglobinuria Treatment Danicopan First Approval Received in Japan

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AstraZeneca (AZ) has achieved a significant milestone with the first global regulatory approval for Voydeya, an oral Factor D inhibitor, in Japan, representing a breakthrough in the treatment of paroxysmal nocturnal hemoglobinuria (PNH). Voydeya is licensed as an add-on therapy to complement C5 inhibitors like AZ’s own Soliris (eculizumab) and Ultomiris (ravulizumab), specifically targeting patients who do not respond adequately to C5 drugs alone.

PNH is a condition where the body’s complement system attacks red blood cells, causing anemia, fatigue, and in severe cases, necessitating regular blood transfusions. While C5 inhibitor treatment is generally effective, some patients experience a phenomenon known as extravascular hemolysis (EVH), where changes in red blood cells lead to their destruction in the spleen, bone marrow, or liver, resulting in continued symptoms. Danicopan has been developed to address this issue in the estimated 10% to 20% of paroxysmal nocturnal hemoglobinuria patients who suffer from EVH.

AZ Enhances Paroxysmal Nocturnal Hemoglobinuria Lineup with Danicopan Amid Growing Pharma Competition

The acquisition of danicopan was part of AZ’s $39 billion deal to buy Alexion in 2021, which also included Soliris and Ultomiris. This approval is seen as a strategy to bolster AZ’s paroxysmal nocturnal hemoglobinuria franchise, especially as it begins to face competition from other pharmaceutical companies. Competitors include Novartis with its oral targeted factor B inhibitor Fabhalta (iptacopan), Apellis Pharmaceuticals’ C3 inhibitor Empaveli (pegcetacoplan), administered via subcutaneous infusion, and Roche’s subcutaneous C5 candidate crovalimab. Meanwhile, Soliris is expected to lose patent protection starting the following year.

Despite facing competition, AZ’s Soliris and Ultomiris have performed differently in the market. In the first nine months of the year, Soliris sales decreased by 15% to $2.43 billion, while Ultomiris sales grew 58% to $2.14 billion, aided by its less frequent dosing requirements.

Paroxysmal Nocturnal Hemoglobinuria

Japan’s MHLW Approves Voydeya for PNH Following Positive ALPHA Trial Results

The approval of Voydeya by the Japanese Ministry of Health, Labour, and Welfare (MHLW) is grounded in the positive results of the ALPHA phase 3 trial. The trial demonstrated sustained improvements in hemoglobin levels and reduced markers of red blood cell destruction over a 48-week follow-up period. Marc Dunoyer, the chief executive of Alexion, remarked on the more than two decades of paroxysmal nocturnal hemoglobinuria research that has established the effectiveness of C5 inhibition in treating this rare disease. He emphasized that Voydeya, as an add-on to standard care, reflects the company’s commitment to addressing the needs of patients impacted by clinically significant EVH without disrupting their current successful therapy.

Looking forward, AZ anticipates responses from European and US regulators regarding danicopan in the first half of the year. Additionally, the drug is being tested in a phase 2 trial for geographic atrophy, a leading cause of vision loss. If successful, this could significantly expand the market potential for danicopan, marking a new era in the treatment of both PNH and other potential indications. This milestone underscores AstraZeneca’s ongoing commitment to innovation and addressing unmet medical needs in rare diseases.


Resource: Pharmaphorum, January 18, 2024

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