PNH treatment Voydeya available soon at low or no cost in England
Add-on therapy can prevent red blood cell destruction outside blood vessels
England’s National Institute for Health and Care Excellence (NICE) has recommended that the oral treatment Voydeya (danicopan) be covered by the country’s National Health Service (NHS) and provided at low or no cost to certain adults with paroxysmal nocturnal hemoglobinuria (PNH).
The recommendation covers Voydeya’s use as an add-on to standard treatment with C5 inhibitors Ultomiris (ravulizumab) or Soliris (eculizumab) in PNH patients with residual anemia due to significant extravascular hemolysis (EVH), or red blood cell destruction outside blood vessels.
All three therapies are marketed by Alexion, AstraZeneca Rare Disease.
“Today’s recommendation from NICE is a positive milestone for eligible individuals with PNH who experience the added burden of EVH symptoms such as continued [anemia], while treated with a C5 inhibitor,” Morag Griffin, a consultant hematologist at Leeds Teaching Hospitals NHS Trust, in the U.K., said in a press release.
NICE’s recommendation is expected to be implemented in the next few months. It will not affect current treatment with Voydeya in England. People receiving treatment outside this recommendation may continue without changing previously established funding arrangements.
The NHS in Wales will also follow NICE’s recommendation.
Add-on PNH treatment reduces red blood cell destruction outside blood vessels
PNH is marked by hemolysis, the destruction of red blood cells, due to the abnormal activation of the complement system. The group of immune proteins that make up this system normally help antibodies and immune cells clear microbes and damaged cells from the body.
Ultomiris and Soliris are standard PNH treatments designed to block complement activation by targeting a complement protein called C5. Although these therapies effectively prevent hemolysis within blood vessels (intravascular hemolysis), some patients have persistent PNH symptoms due to EVH.
Voydeya works by blocking another complement protein called factor D. By blocking this protein, the therapy can reduce EVH without interfering with C5 inhibitors’ effects on intravascular hemolysis.
It is approved in the U.K. as an add-on to Ultomiris or Soliris for treating adult PNH patients with residual hemolytic anemia, or low red blood cells due to hemolysis. It’s also approved for similar indications in the European Union, the U.S., Canada, and Japan.
Regulatory approvals were based on data from the Phase 3 ALPHA clinical trial (NCT04469465), which evaluated the add-on therapy against a placebo in 86 patients with substantial EVH despite treatment with Ultomiris or Soliris.
The ALPHA trial investigated the safety and efficacy of [Voydeya] in patients with PNH and EVH, and demonstrates this is a suitable option to support disease control, and may help to improve patients’ quality of life.
The results showed that Voydeya outperformed the placebo in easing anemia and lowering the need for blood transfusions, with sustained efficiency seen for up to a year of treatment.
The most common adverse events with the therapy in ALPHA were headache, nausea, joint pain, and diarrhea. Because it suppresses part of the immune system, Voydeya can increase the risk of serious infections.
“The ALPHA trial investigated the safety and efficacy of [Voydeya] in patients with PNH and EVH, and demonstrates this is a suitable option to support disease control, and may help to improve patients’ quality of life,” Griffin said.
Voydeya will be available for free or with a small copay to eligible patients
NICE is responsible for drafting recommendations about which therapies will be listed for public reimbursement and who will be eligible for them. Recommendations are based on a treatment’s potential benefits along with its cost, which is determined in negotiations between the NHS and the therapy’s developer.
Voydeya’s list price is £1,369.80 (about $1,780) for a 90‑tablet bottle of 50 mg tablets and £2,739.60 (about $3,560) for a 90‑tablet bottle of 100 mg tablets. Its listing on the NHS means the therapy will be available for free or with a small copay to eligible patients. Terms of the commercial agreement between NHS England and Alexion are confidential and the agreed price was not disclosed.
Deborah Richards, general manager at Alexion, noted that Voydeya “is the latest innovation in decades of work in our complement inhibition research programme.”
“We’re committed to bettering the lives of all patients with PNH and we look forward to making this treatment available to eligible patients across the country,” Richards said.
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