26 February 2024
Voydeya recommended for approval in the EU by CHMP as add-on treatment to ravulizumab or eculizumab for adults with PNH who have residual haemolytic anaemia
Recommendation of first-in-class, oral, Factor D inhibitor based on ALPHA Phase III trial results
Voydeya (danicopan) has been recommended for marketing authorisation in the European Union (EU) as an add-on to ravulizumab or eculizumab for the treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH) who have residual haemolytic anaemia. Voydeya is a first-in-class, oral, Factor D inhibitor developed as an add-on to standard-of-care Ultomiris (ravulizumab) or Soliris (eculizumab) to address the needs of the approximately 10-20% of patients with PNH who experience clinically significant extravascular haemolysis (EVH) while treated with a C5 inhibitor.1,2
The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) 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.1
PNH is a rare and severe blood disorder characterised by the destruction of red blood cells within blood vessels, known as intravascular haemolysis (IVH), and white blood cell and platelet activation that can cause thrombosis (blood clots) and result in organ damage and potentially premature death.3-5 Immediate, complete and sustained terminal complement inhibition by blocking the C5 protein helps reduce symptoms and complications, resulting in improved survival for patients with PNH.5-8 Approximately 10-20% of people living with PNH who are treated with a C5 inhibitor experience clinically significant EVH, which can result in continued symptoms of anaemia and require blood transfusions.1-3,9-11
Professor Hubert Schrezenmeier, MD, Medical Director, Institute of Transfusion Medicine at The University of Ulm, said: “C5 inhibition with Ultomiris or Soliris is the standard-of-care in PNH, proven to control IVH and reduce life-threatening thrombotic events, yet a small portion of patients may experience clinically significant EVH. In the ALPHA trial, Voydeya as an add-on to Soliris or Ultomiris increased haemoglobin levels and reduced fatigue, anaemia and transfusion dependence. If approved, Voydeya may optimise care for people impacted by this burdensome condition while allowing patients to maintain disease control with an established C5 inhibitor.”
Marc Dunoyer, Chief Executive Officer, Alexion, said: “Today’s 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.”
The ALPHA Phase III trial evaluated the efficacy and safety of Voydeya as an add-on to Ultomiris or Soliris in patients with PNH who experienced clinically significant EVH. Results showed that Voydeya met the primary endpoint of change in haemoglobin from baseline to week 12 and all key secondary endpoints, including transfusion avoidance and change in Functional Assessment of Chronic Illness Therapy – Fatigue (FACIT-Fatigue) score.1
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.1
Voydeya has been granted Breakthrough Therapy designation by the US Food and Drug Administration 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.