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FDA approves AstraZeneca's drug for rare disease

Bio Tech

FDA approves AstraZeneca drug rare disease

Voydeya, a drug acquired by Alexion, has been approved as an additional treatment for individuals with the blood disorder paroxysmal nocturnal hemoglobinuria.

For those suffering from the uncommon and dangerous blood condition known as paroxysmal nocturnal hemoglobinuria, or PNH, the Food and Drug Administration has approved a novel medication.

AstraZeneca's Voydeya is a medication that can be used as a supplemental therapy in addition to Ultomiris and Soliris, the company's existing PNH therapies. It is intended for the 10% to 20% of PNH patients who, in spite of receiving those additional medications, nonetheless suffer from severe "hemolysis," or the early breakdown of red blood cells.

The approval strengthens the company that AstraZeneca acquired for $39 billion in 2020 when it acquired Alexion Pharmaceutical. Because Ultomiris and Soliris are approved for PNH as well as other illnesses, that deal made AstraZeneca a player in rare disease research. The two medications rank among AstraZeneca's best-selling items, with combined revenues expected to reach over $6 billion in 2023.

Additionally, Alexion was developing medications like Voydeya to stave off competition from businesses like Novartis, Roche, and Amgen. When the business acquired Achillion Pharmaceuticals for around $1 billion in 2019, it also acquired Voydeya, which was formerly known as Danicopan.

Voydeya, like Ultomiris and Soliris, targets the complement system, an innate immune system component linked to a number of illnesses, including PNH. However, Voydeya is not an IV infusion; it is a tablet. Additionally, unlike current treatments, it inhibits Factor D, a distinct component of the complement system.

Experiments revealed that, in comparison to normal therapy alone, the combination of Voydeya and C5 inhibitors such as Ultomiris and Soliris raised levels of oxygen-carrying hemoglobin and decreased the need for blood transfusions. Headache, joint discomfort, diarrhea, and nausea were the most frequent adverse effects. The Lancet Hematology reported the results last year.

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