Alexion, AstraZeneca Rare Disease has received approval from the US Food and Drug Administration (FDA) for Voydeya (danicopan) as an add-on therapy to treat extravascular haemolysis (EVH) in adults with paroxysmal nocturnal haemoglobinuria (PNH).

A first-in-class, oral Factor D inhibitor, Voydeya has been developed for patients who continue to suffer from EVH despite treatment with C5 inhibitors such as Ultomiris (ravulizumab) or Soliris (eculizumab).

The FDA’s decision is grounded in the positive outcomes of the ALPHA Phase III trial, which demonstrated that Voydeya met its primary endpoint of haemoglobin change from baseline to week 12, alongside all key secondary endpoints.

The drug was generally well-tolerated with no new safety concerns.

As part of the ALPHA double-blind, placebo-controlled, multiple-dose trial, patients were enrolled and randomised to receive Voydeya or placebo apart from their ongoing Soliris or Ultomiris therapy over 12 weeks.

It aimed to assess the superiority of Voydeya as an adjunct to C5 inhibitor therapy.

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The trial included a prespecified interim analysis and an option for a two-year extension period for those completing the initial 24 weeks.

Japan already approved Voydeya and the European Union is poised to follow suit.

The drug has also achieved breakthrough therapy designation in the US and PRIority MEdicines (PRIME) status in the EU.

Alexion CEO Marc Dunoyer stated: “The approval of first-in-class, Factor D inhibitor Voydeya marks an important advancement in the treatment of PNH and builds on our leadership and commitment to bring forward innovation in complement science.

“As the ALPHA trial suggests, dual complement pathway inhibition at Factor D and C5 may be an optimal treatment approach for this subset of patients with EVH, enabling them to continue with proven standard-of-care therapy.”

Alexion is also exploring Voydeya’s potential as a monotherapy for geographic atrophy in a Phase II clinical trial.

The FDA recently approved Alexion’s Ultomiris to treat adults with anti-aquaporin-4 antibody-positive neuromyelitis optica spectrum disorder.