The Food and Drug Administration has approved a first-of-its-kind drug for people with the rare and serious blood disease paroxysmal nocturnal hemoglobinuria, or PNH.
Called Voydeya and owned by AstraZeneca, the drug is cleared for use as an add-on therapy to the standard PNH treatments, Ultomiris and Soliris, the pharmaceutical company already sells. It’s meant for the estimated 10% to 20% of people with PNH who still experience significant “hemolysis,” or premature destruction of red blood cells, despite treatment with those other drugs.
The approval builds up a business AstraZeneca inherited when it bought Alexion Pharmaceutical for $39 billion in 2020. That deal established AstraZeneca as a player in rare disease research due to Ultomiris and Soliris, which are each approved for PNH as well as other conditions. The two drugs are among AstraZeneca’s top-selling products, generating more than $6 billion in combined sales in 2023.
Alexion also had drugs in its pipeline, such as Voydeya, that were meant to defend against rising competition from companies like Novartis, Roche and Amgen. The company had acquired Voydeya, previously known as danicopan, when it bought Achillion Pharmaceuticals for nearly $1 billion in 2019.
Like Ultomiris and Soliris, Voydeya targets the complement system, a part of the innate immune system that’s implicated in a variety of diseases including PNH. But Voydeya is a pill, rather than an intravenous infusion. It also blocks a different part of the complement system, Factor D, than existing therapies.
Testing showed a combination of Voydeya and C5 inhibitors like Ultomiris and Soliris increased levels of oxygen-carrying hemoglobin and reduced the need for blood transfusions compared to standard therapy alone. The most common side effects were headache, joint pain, diarrhea and nausea. Results were published in The Lancet Hematology last year.
“The approval of first-in-class, Factor D inhibitor Voydeya marks an important advanced in the treatment of PNH and builds on our leadership and commitment to bring forward innovation in complement science,” said Alexion CEO Marc Dunoyer, in a Monday statement. The drug is already available in Japan.
Jefferies analyst Peter Welford has estimated $750 million in peak annual worldwide sales for Voydeya in PNH. AstraZeneca is also testing the drug in the eye disease geographic atrophy. A Phase 2 trial is ongoing.
Through Alexion, AstraZeneca aims to extend its reach into other rare diseases, too. For instance, since the Alexion deal, AstraZeneca has bulked up its drug portfolio through licensing deals and buyouts focused on multiple forms of amyloidosis. It’s also made forays into genetic medicine and cell therapy.