healthcare professional
In the superiority Phase 3 ALPHA trial, the safety and efficacy of VOYDEYA® was evaluated based on 12-week data from 63 adult patients with Paroxysmal nocturnal hemoglobinuria (PNH) who received VOYDEYA® or placebo at the recommended dosing regimen as add-on therapy to ULTOMIRIS® (ravulizumab) or SOLIRIS® (eculizumab).1
Visit the Study Design page to get more information on the study design of the ALPHA trial.
Visit the Study Design page to get more information on the study design of the ALPHA trial.
Primary endpoint met: Patients who received VOYDEYA® achieved a clinically meaningful, statistically significant increase in mean Hb levels from baseline compared with placebo at 12 weeks (change in least squares mean [LSM] Hb: 2.94 [95% CI 2.52 to 3.36] g/dL versus 0.50 [95% CI -0.13 to 1.12] g/dL; p<0.0001).*†1,2
*Based on MMRM test.1,2
†A change in Hb of ≥2 g/dL is considered clinically meaningful.1
†A change in Hb of ≥2 g/dL is considered clinically meaningful.1
of patients treated with VOYDEYA® were transfusion-free* over 12 weeks, compared with 38% (8/21) of patients treated with placebo (p=0.0004).1
of patients receiving VOYDEYA® were transfusion free* at Week 72 compared with 8.8% (5/57; VOYDEYA® group) and 17.2% (5/29; placebo group) in the 24 weeks prior to screening.3
of patients treated with VOYDEYA® had a Hb increases of ≥2 g/dL† at 12 weeks in the absence of transfusion compared with 0% (0/21) of patients treated with placebo (p<0.0001),1 a similar treatment effect was observed over 72 weeks.3
Patients treated with VOYDEYA® had significantly reduced absolute reticulocyte count (ARC) from baseline at Week 12 compared with placebo (–83.8×109/L [95% CI: –101.6 to –65.9] vs 3.5×109/L [95% CI: –21.9 to 28.8]; p<0.0001) and levels were maintained to Week 72.3
*Patients who did not receive a transfusion and did not meet the protocol-specified guidelines for transfusion through the 12-week treatment period were considered to have been transfusion free.1
†A change in haemoglobin of ≥2 g/dL is considered clinically meaningful.1
†A change in haemoglobin of ≥2 g/dL is considered clinically meaningful.1
Adding VOYDEYA® to ULTOMIRIS® or SOLIRIS® resulted in significantly improved FACIT-Fatigue scores* at Week 12 compared with placebo (8.0 points [95% CI: 5.7 to 10.2] vs 1.9 points [95% CI: –1.3 to 5.0]; p=0.0021).1
Mean FACIT-Fatigue scores observed with VOYDEYA® at Week 12 were comparable to those reported in the general population and were sustained to Week 72†.3
Mean FACIT-Fatigue scores observed with VOYDEYA® at Week 12 were comparable to those reported in the general population and were sustained to Week 72†.3
*Fatigue was self-assessed using the FACIT-Fatigue scale.5,6 A change in FACIT-Fatigue score of ≥5 points is considered clinically meaningful in PNH.5
†Mean FACIT-Fatigue score for the general population was determined through assessment of 2,426 adults (n=1,074 males; n=1,352 females; mean age [SD]: 49.8 [17.4] years) in Germany between March 2015 and May 2015.6
†Mean FACIT-Fatigue score for the general population was determined through assessment of 2,426 adults (n=1,074 males; n=1,352 females; mean age [SD]: 49.8 [17.4] years) in Germany between March 2015 and May 2015.6
IVH control (as evaluated by lactose dehydrogenase [LDH] levels of <1.5xULN*) was maintained at Week 72 in both arms, through long-term control of terminal complement activity via C5 inhibition.3
- 86% (6/7) of events were mild to moderate in severity and all resolved rapidly without trial discontinuation, dose adjustment or need for transfusion.3
- One event was associated with LDH ≥2x ULN (2.2x ULN) and decreased Hb, which was temporarily associated with COVID-19 infection.3
*ULN for LDH levels in clinical trials of ULTOMIRIS® in PNH was taken to be 246 U/L.7,8
VOYDEYA® was well-tolerated over 12 weeks in the clinical trial and in long-term follow up to Week 721-3
Find out more by visiting the VOYDEYA® safety page below.
Safety 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; C5, complement component 5; CI, confidence interval; EVH, extravascular haemolysis; FACIT, Functional Assessment of Chronic Illness Therapy; IVH, intravascular haemolysis; LDH, lactate dehydrogenase; LSM, least squares mean; PNH, paroxysmal nocturnal haemoglobinuria; SD, standard deviation; SE, standard error; ULN, upper limit of normal; Hb, haemoglobin.