healthcare professional
To assess the efficacy and safety of VOYDEYA® – a first-in-class, oral complement factor D inhibitor – as add-on therapy to ULTOMIRIS® (ravulizumab) or SOLIRIS® (eculizumab) in adult patients with Paroxysmal nocturnal hemoglobinuria (PNH) and clinically significant extravascular haemolysis (cs-EVH)*.1
VOYDEYA® was studied in a randomised, double-blind, placebo-controlled superiority Phase 3 study in which adult patients with PNH and cs-EVH received either VOYDEYA® (n=49) or placebo (n=24) in addition to their stable dose (≥6 months) of ULTOMIRIS® or SOLIRIS®.1,2
After the randomised treatment period of 12 weeks (TP1), patients receiving placebo were switched to VOYDEYA® and patients in the VOYDEYA® arm continued treatment to Week 24 (TP2).1
A prespecified interim efficacy analysis was performed when 63 patients reached the end (either completed or discontinued) of the 12-week treatment period†.1
A prespecified interim efficacy analysis was performed when 63 patients reached the end (either completed or discontinued) of the 12-week treatment period†.1
Inclusion and exclusion criteria1
Overall, the disease characteristics at baseline were generally balanced between the two treatment groups.1
Baseline disease characteristics such as haemoglobin (Hb), absolute reticulocyte count (ARC) and FACIT-Fatigue scores were indicative of cs-EVH*.1
At baseline, Patients with low Hb and increased ARC had well-controlled intravascular haemolysis with lactate dehydrogenase (LDH) levels of <1.5× ULN‡.1
Baseline disease characteristics such as haemoglobin (Hb), absolute reticulocyte count (ARC) and FACIT-Fatigue scores were indicative of cs-EVH*.1
At baseline, Patients with low Hb and increased ARC had well-controlled intravascular haemolysis with lactate dehydrogenase (LDH) levels of <1.5× ULN‡.1
Other secondary efficacy endpoints were the proportion of patients with a haemoglobin increase of ≥2 g/dL at Week 24 in the absence of transfusion, the change in the number of red blood cells transfused and transfusion instances at Week 12 and Week 24, the change from baseline in total and direct bilirubin, PNH red blood cell clone size, C3 fragment disposition on PNH red blood cells, LDH at Week 12, the percentage of participants with haemoglobin normalisation (defined as haemoglobin value above the lower limit of normal) at Week 12 and the change from baseline in FACIT-Fatigue scores at Week 24.
*Defined in this study as Hb ≤9.5 g/dL and ARC ≥120×109/L with or without transfusion.1
†As of the interim analysis cut-off date (28 June 2022), 73 participants had been randomised, 61 participants completed TP1,and 2 participants discontinued TP1 (one from each arm) due to treatment-emergent adverse events (TEAEs).1
‡LDH reference range: 135–330 U/L.3
§Defined as remaining transfusion-free without requirement for transfusion per protocol-specified guidelines [haemoglobin <7 g/dL regardless of clinical signs or symptoms or haemoglobin <9 g/dL in the presence of clinical signs or symptoms] through to Week 12.1
Add-on VOYDEYA® has shown clinically meaningful improvements in Hb and fatigue at Week 12, with improvements in both maintained to Week 721-3
Detailed information on the efficacy of Voydeya® is available below.
Efficacy of VOYDEYA®
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/
ARC, absolute reticulocyte count; EVH, extravascular haemolysis; Hb, haemoglobin LTE; long-term extension; SD, standard deviation; TP, treatment period; TEAE, treatment-emergent adverse event. PNH, cs = clinical significant, LDH, Lactate Dehydrogenase; FACIT, Functional Assessment of Chronic Illness Therapy: C5, C5 Inhibitors; ULN, upper limit of normal.