FDA approves Voydeya as add-on therapy for adults with PNH

List price pending for add-on oral treatment to Ultomiris or Soliris

Marisa Wexler MS avatar

by Marisa Wexler MS |

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The U.S. Food and Drug Administration (FDA) has approved Voydeya (danicopan) as an add-on treatment for extravascular hemolysis, or the destruction of red blood cells taking place outside blood vessels, in adults with paroxysmal nocturnal hemoglobinuria (PNH).

The treatment is to be taken with the standard-of-care therapies Ultomiris (ravulizumab) and Soliris (eculizumab).

Voydeya’s approval in the U.S. came a few months after the medication was approved in Japan for the same indication. The therapy is also up for approval in Europe.

“The approval of … Voydeya marks an important advancement in the treatment of PNH and builds on our leadership and commitment to bring forward innovation in complement science,” Marc Dunoyer, CEO of Alexion, AstraZeneca Rare Disease, which developed Voydeya, said in a company press release.

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Voydeya expected to help control symptoms in up to 20% of patients with EVH

The newly approved therapy is expected to help control disease symptoms in the 10% to 20% of PNH patients who experience substantial EVH despite treatment with standard-of-care medications, according to Alexion, which also developed Ultomiris and Soliris.

“The approval of Voydeya offers this small subset of PNH patients an add-on therapy designed to address EVH, while maintaining disease control with Ultomiris or Soliris,” said Bart Scott, MD, a professor at the University of Washington.

In PNH, part of the immune system called the complement cascade becomes abnormally activated, leading to hemolysis or the destruction of red blood cells. Soliris and Ultomiris both work to block the activity of the complement system by targeting a protein called C5. This is generally effective for stopping hemolysis that occurs within blood vessels, which is also known as intravascular hemolysis (IVH). Yet, some PNH patients on these medicines continue experiencing notable disease symptoms due to persistent EVH.

Voydeya is a first-in-class oral therapy that’s designed to stop EVH without interfering with the ability of Soliris and Ultomiris to control IVH. It does so by blocking the activity of another complement protein called factor D.

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Voydeya supplied in 50 or 100 mg oral tablets

Voydeya is supplied in the form of oral tablets containing 50 or 100 mg of the therapy’s active ingredient, which can be taken with or without food. Its recommended starting dosage is 150 mg three times a day, which can be increased to 200 mg three times a day, depending on a patient’s clinical response.

The FDA’s approval of Voydeya was based mainly on data from a Phase 3 clinical trial called ALPHA (NCT04469465), which tested Voydeya against a placebo as an add-on treatment in 86 adults with PNH who were experiencing substantial EVH while on Soliris or Ultomiris.

In the first three months of the study, patients were randomly assigned to receive either Voydeya (at an initial dose of 150 mg, which could be increased to 200 mg) or a placebo, given on top of standard treatment. After that, all patients received add-on treatment with Voydeya for another period of approximately three months. Patients who completed both treatment periods in the trial were eligible to enter a long-term extension phase to continue receiving add-on Voydeya treatment for two years.

Results from the first three months of the study indicated Voydeya significantly increased the levels of hemoglobin — the protein that red blood cells use to ferry oxygen through the bloodstream — compared with the placebo. Voydeya also outperformed the placebo in easing fatigue and reducing the need for blood transfusions.

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Voydeya’s positive effects on hemoglobin sustained for up to 1 year

More recent data from the study also indicated Voydeya’s positive effects on hemoglobin were sustained through the second three-month treatment period and for up to about one year in total.

“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,” Dunoyer said.

The most common side effects of Voydeya reported in the ALPHA trial included headache, nausea, joint pain, and diarrhea. Voydeya’s prescribing information also comes with a boxed warning noting the therapy can increase a patient’s risk of developing serious and life-threatening infections caused by certain species of encapsulated bacteria, including the one causing meningitis.

In its announcement, Alexion did not specify the expected list price of Voydeya. A recent analysis from an independent nonprofit suggested a price of $12,300 to $13,100 per year would be reasonable given the expected benefits of Voydeya when used as an add-on treatment for patients on Soliris or Ultomiris.