AstraZeneca claims positive results for its “add-on” drug for rare blood disease

AstraZeneca on Friday announced its experimental “add-on” drug for people with paroxysmal nocturnal hemoglobinuria succeeded in a Phase 3 study. 

The drug candidate, called ALXN2040, is meant to complement AstraZeneca’s drugs Ultomiris and Soliris, which are already approved to treat the rare blood disorder. 

PNH is a rare disease that can be severe, causing the destruction of blood cells and potentially leading to blood clots.

ALXN2040 is for people with PNH who also experience extravascular hemolysis, which refers to the elimination of red blood cells outside of blood vessels. Extravascular hemolysis is sometimes experienced by patients with PNH who receive treatments like Ultomiris and Soliris, and can lead to anemia or require blood transfusions.

In its Friday statement, AstraZeneca said ALXN2040 met its primary goal in its Phase 3 study, claiming the drug helped study participants avoid transfusions and improved scores on a disease rating scale. When taken with Ultomiris or Soliris, ALXN2040 resulted in improved hemoglobin levels compared to a placebo, according to the statement. 

AstraZeneca didn’t disclose any specific data in statement, but said it would present some in the future. The company plans to seek approval from regulators within the coming months. 

AstraZeneca acquired the drug through its 2020 purchase of Alexion Pharmaceuticals, which had itself bought the medicine via a 2019 deal for Achillion Pharmaceuticals.

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