AstraZeneca's blood-disorder drug recommended for EU approval

The Committee for Medicinal Products for Human Use of the European Medicines Agency said Voydeya, whose generic name is danicopan, has been recommended for marketing authorisation as an add-on to ravulizumab or eculizumab for the treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH), a rare and severe blood disorder which destroys red blood cells and can lead to blood clots and organ damage.

The positive opinion was based on results of the 12-week ALPHA Phase III trial, results of which were published in The Lancet in December.

Marc Dunoyer, chief executive officer of AstraZeneca's rare disease division Alexion, said the welcome news "recognises the promise of Voydeya as an add-on to standard-of-care to address signs and symptoms of clinically significant EVH for this small subset of patients".

Voydeya is an oral, Factor D inhibitor developed as an add-on to standard-of-care Ultomiris (ravulizumab) or Soliris (eculizumab) to address the needs of the around 10-20% of patients with PNH who experience clinically significant extravascular haemolysis (EVH) while treated with a C5 inhibitor, AstraZeneca said.

"As we saw in the pivotal ALPHA Phase III trial, dual complement pathway inhibition at Factor D and C5 may be an optimal treatment approach for these patients," Dunoyer said.