Safety and Tolerability Profile of VOYDEYA® (danicopan)
The safety and tolerability profile of VOYDEYA® was derived from 12-week data for 73 adult patients with Paroxysmal nocturnal haemoglobinuria (PNH) who received VOYDEYA® or placebo at the recommended dosing regimen as add-on therapy to ULTOMIRIS® (ravulizumab) or SOLIRIS® (eculizumab) in the Phase 3 ALPHA study.1

Visit the Study Design page to get more information on the study design of the ALPHA trial.
 ALPHA Study Design
*There were no meningococcal infections or discontinuations due to haemolysis over 72 weeks of treatment.3 Treatment-related serious adverse events were reported in two patients (pancreatitis and increased blood bilirubin in one patient by Week 12 and headache in the other by Week 24), and no further treatment-related serious adverse events were observed over long-term follow-up to Week 72.3

†Hepatic enzyme increased’ includes preferred terms alanine aminotransferase increased, hepatic function abnormal, hepatic enzyme increased, and transaminases increased. None of the liver enzyme elevations was serious.1,2
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Add-on VOYDEYA® has shown clinically meaningful†¶ improvements in haemoglobin and fatigue at Week 12, with improvements in both maintained through to Week 72.1–3
Detailed information on the efficacy of Voydeya® is available below.
Efficacy of VOYDEYA®
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†Fatigue was self-assessed using the FACIT-Fatigue scale. A change in FACIT-Fatigue score of ≥5 points is considered clinically meaningful in PNH.
A change in haemoglobin of ≥2 g/dL is considered clinically meaningful.

Adverse Event Reporting

Please report any adverse events via your national reporting system. Adverse events can also be reported to Alexion,
AstraZeneca Rare Disease by contacting: https://contactazmedical.astrazeneca.com/
Lee JW, et al. . Addition of Danicopan to Ravulizumab or Eculizumab in Patients with Paroxysmal Nocturnal Haemoglobinuria and Clinically Significant Extravascular Haemolysis (ALPHA): a Double-blind, Randomised, Phase 3 Trial. The Lancet Haematology. 2023;10:e955 – e965. VOYDEYA® EU Summary of Product Characteristics available at: https://www.ema.europa.eu/en/documents/product-information/voydeya-epar-product-information_en.pdf. Last accessed November 2025. Kulasekararaj A, et al. Long-term Efficacy and Safety of Danicopan as Add-on Therapy to Ravulizumab or Eculizumab in PNH with Significant EVH. Blood. 2025;145(8):811–822. advance I am a healthcare professional registered in the EU I am not a
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