EU okays AstraZeneca’s factor D inhibitor Voydeya for rare blood disorder

2024-04-23

临床结果临床3期上市批准

The European Commission approved AstraZeneca’s Voydeya (danicopan) as an add-on treatment for adults with paroxysmal nocturnal haemoglobinuria (PNH) receiving C5 inhibitor therapies Ultomiris (ravulizumab) or Soliris (eculizumab). The drugmaker noted Tuesday that the factor D inhibitor specifically addresses significant extravascular haemolysis (EVH), a phenomenon seen in around 10% to 20% of C5 inhibitor C5 inhibitor recipients.

Hot on the heels of recent Japanese and US approvals, the decision follows a positive stance adopted by the European Medicines Agency's (EMA) drug advisory body in February. “We look forward to making this first-in-class factor D inhibitor available to patients in Europe and to advancing access around the globe,” remarked Marc Dunoyer, CEO of the company’s rare disease unit Alexion.

The EU approval was based on positive findings from the Phase III ALPHA study, which investigated add-on Voydeya in Ultomiris- or Soliris-treated patients with PNH and clinically significant EVH. Results showed that Voydeya met the primary endpoint of change in haemoglobin from baseline to week 12, alongside all key secondary goals. Moreover, the therapy was well tolerated, with no new safety signals observed.

Another drug on the brink of EU approval in PNH is Novartis' factor B inhibitor Fabhalta (iptacopan), which recently secured a positive opinion from the EMA’s Committee for Medicinal Products for Human Use.

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