The United States Food and Drug Administration (USFDA) has given a nod to AstraZeneca’s Voydeya (danicopan) to treat paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. The US regulatory body has signed off on the factor D inhibitor specifically with extravascular hemolysis (EVH).
In this condition, the red blood cells (RBCs) get destroyed outside the blood vessels. According to a report by Fierce Pharma, the nod comes three months after the Japanese Ministry of Health, Labour and Welfare (JHLW) became the first regulator worldwide to endorse the therapy.
It is noteworthy that AstraZeneca has been a key player in the therapeutic area. However, Novartis’ Fabhalta recently got approval for the disorder. Reportedly, to keep its dominance in the market, AstraZeneca has introduced its add-on oral treatment in the United States. The company made the announcement of the drug approval on Monday.
According to a report by Fierce Pharma, Voydeya acts as an add-on for the company’s pair of PNH standard-of-care blockbusters—Soliris, which has been on the market for 17 years and generated $3.2 billion in sales in 2023, and its follow-on Ultomiris, which was approved in 2018 and accounted for $3 billion in sales last year.
Bart Scott, MD, Professor, Division of Hematology and Oncology at the University of Washington Medical Center, and Professor, Clinical Research Division at Fred Hutchinson Cancer Center, said in a company statement on Monday: “The approval of Voydeya offers this small subset of PNH patients an add-on therapy designed to address EVH, while maintaining disease control with Ultomiris or Soliris. Terminal complement inhibition with Ultomiris can address the life-threatening complications of PNH, building on the efficacy and safety of Soliris established over nearly 20 years.”
In 2021, AstraZeneca gained the drug in its acquisition of Alexion for $39 billion. In 2019, Alexion picked up the drug in $930 million buyout of another Connecticut company, Achillion.
In February this year, Europe’s regulator recommended Voydeya for approval. Moreover, AZ also is investigating danicopan as a treatment for the macular degeneration condition geographic atrophy.
