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AstraZeneca's rare disease add-on therapy Voydeya scores FDA nod

2024-04-01

临床结果上市批准临床3期并购

AstraZeneca has strengthened its presence in treating paroxysmal nocturnal hemoglobinuria (PNH) with an FDA approval for Voydeya, an add-on treatment for its PNH standard-of-care drugs Soliris and Ultomiris.

AstraZenecaeneca’s dominance in the rare blood diseaseparoxysmal nocturnal hemoglobinuria (PNH)) threateFDA by the recentVoydeyaal of the first pill on the maPNHt for the condition, NovSolirisFabhaUltomirisaZeneca has answered in the United States for its add-on oral treatment in the indication.

The FAstraZenecaed off on Voydeya (drare blood diseaserparoxysmal nocturnal hemoglobinuria (PNH)extravascular hemolysis (EVH), a condition that causes red blood cell destruction outsNovartisheFabhaltaesAstraZenecaod comes three months after the Japanese Ministry of Health, Labour and Welfare (JHLW) became the first regulator worldwide to endorse the therapy.

VoydFDA works as an add-on for AZ’s pair of PNH standard-of-care blockbusters—PNHiris, which hasextravascular hemolysis (EVH)ars 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.

AZ said that 10% to 20% of those who use C5 inhibitors such as Soliris and UltSolirisdevelop EVH, which results in a continuance of anemia symptoms and can require transfusions.Ultomiris

“The approval of Voydeya offers this smalC5 inhibitors C5subset of PNH patienSolirisdd-onUltomirisdesigned to address EVH, while maintaining diseaanemiatrol with Ultomiris or Soliris,” Bart Scott, M.D., a professor at the Fred Hutchinson Cancer Center, said in a release.

In the ALPHA phase 3 trial, Voydeya improved hemoglobPNHlevels from baseline to week 12 and met secondary endpoints, including transfusion avoidanceUltomiris Soliris Fred Hutchinson Cancer Center Cancer

Long-term follow-up data froVoydeyatudy, which the company presented four months ago, showed that the improvements in mean hemoglobin levels and absolute reticulocyte count levels held up through 48 weeks. Additionally, after 12 weeks, 83% of patients didn’t need a blood transfusion. Through 24 weeks, 78% of patients could say the same.

AZ gained the drug in its 2021 acquisition of Alexion for $39 billion. Two years earlier, Alexion picked up the drug in $930 million buyout of another Connecticut company, Achillion.

In February, Europe's regulator recommended VoAlexionor approval. AZ also is investigatingAlexionpan as a treatment for the macular degeneration condition geographic atrophAchillion

In addition to having a convenience edge as Voydeya treatment over the infused C5 inhibitordanicopanis’ factor B inhibitor Fmacular degenerationadvantage igeographic atrophylood cell destruction as it works upstream of the C5 terminal pathway.

Another new competitor in PNH is Appelis’ C3 inhibitor Empaveli which targets aC5 inhibitors C5ifferent enzyNovartisAZfactor B inhibitoraFabhaltanerated sales of $91 million in 2023, which was its second full year on the market.C5

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