USA — The Food and Drug Administration has granted approval for Voydeya, a groundbreaking medication designed for individuals suffering from the rare and severe blood disorder known as paroxysmal nocturnal hemoglobinuria (PNH), as reported by the media outlet BioPharma.

This landmark approval of AstraZeneca’s Voydeya establishes it as an adjunctive therapy to the standard PNH treatments, Ultomiris and Soliris, already marketed by the pharmaceutical company.

The authorization specifically addresses the needs of the estimated 10% to 20% of individuals with PNH who continue to experience significant “hemolysis,” or premature destruction of red blood cells, despite receiving treatment with these existing drugs.

AstraZeneca’s acquisition of Alexion Pharmaceuticals for US$39 billion in 2020 paved the way for this development, solidifying its position in rare disease research.

Ultomiris and Soliris, approved for PNH and other conditions, have become flagship products for AstraZeneca, generating over US$6 billion in combined sales in 2023.

Inheriting a robust pipeline from Alexion, including Voydeya (previously known as danicopan), AstraZeneca fortified its position against increasing competition from companies like Novartis, Roche, and Amgen.

Unlike its predecessors, Voydeya is administered orally and targets a distinct component of the complement system, Factor D.

Clinical trials have demonstrated that Voydeya, when combined with C5 inhibitors like Ultomiris and Soliris, enhances hemoglobin levels and diminishes the necessity for blood transfusions compared to standard therapy alone.

 Common side effects include headache, joint pain, diarrhea, and nausea, as reported in The Lancet Hematology last year.

Marc Dunoyer, CEO of Alexion, emphasized the significance of Voydeya’s approval as a pioneering Factor D inhibitor, underscoring the company’s dedication to advancing treatment options in complement science.

Voydeya has already been made available in Japan, showcasing its global potential.

Analyst Peter Welford of Jefferies estimates peak annual worldwide sales of $750 million for Voydeya in PNH.

AstraZeneca is also exploring the drug’s efficacy in treating geographic atrophy, with an ongoing Phase 2 trial.

With the Alexion acquisition, AstraZeneca aims to expand its footprint in other rare diseases, leveraging partnerships, acquisitions, and advancements in genetic medicine and cell therapy to bolster its drug portfolio.

For all the latest healthcare industry news from Africa and the World, subscribe to our NEWSLETTER, and YouTube Channel, follow us on Twitter and LinkedIn, and like us on Facebook.