Approval for new Alexion drug to fight rare blood disorder
Following trial results, Voydeya (danicopan) has been recommended for approval in the EU as add-on treatment to existing drugs ravulizumab or eculizumab for adults with Paroxysmal nocturnal hemoglobinuria (PNH) who have residual haemolytic anaemia.
PNH is a rare and severe blood disorder characterised by the destruction of red blood cells within blood vessels, known as intravascular haemolysis and white blood cell and platelet activation that can cause thrombosis and result in organ damage and potentially premature death.
Voydeya is designed to address the needs of the approximately 10-20 per cent of patients with PNH who experience clinically significant extravascular haemolysis while treated with a C5 inhibitor.
The Committee for Medicinal Products for Human Use of the European Medicines Agency based its positive opinion on results from the pivotal ALPHA Phase III trial. Results from the 12-week primary evaluation period of the trial were published in The Lancet Haematology.
Marc Dunoyer, Chief Executive Officer, Alexion, said: “The positive CHMP recommendation 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.
“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.”
Results from the ALPHA Phase III trial showed Voydeya was generally well tolerated and no new safety concerns were identified. In the trial, the most common treatment-emergent adverse events were headache, nausea, arthralgia and diarrhoea.
Voydeya has been granted Breakthrough Therapy designation by the US FDA and PRIority MEdicines (PRIME) status by the EMA. Voydeya has also been granted Orphan Drug Designation in the US, EU and Japan for the treatment of PNH. Voydeya was recently approved in Japan, and regulatory submissions for Voydeya are currently under review in additional countries.
Alexion is also evaluating Voydeya as a potential monotherapy for geographic atrophy in a Phase II clinical trial.
Alexion, AstraZeneca Rare Disease, is the group within AstraZeneca focused on rare diseases, created following the 2021 acquisition of Alexion Pharmaceuticals, Inc.
As a leader in rare diseases for more than 30 years, Alexion is focused on serving patients and families affected by rare diseases and devastating conditions through the discovery, development and commercialisation of life-changing medicines.
• Parent group AstraZeneca has raised new finance through the issue of a $5 billion bond for general corporate purposes, which may include the refinancing of existing debt. Barclays Capital Inc., Citigroup Global Markets Inc., Deutsche Bank Securities Inc. and J.P. Morgan Securities LLC acted as joint book-running managers.
