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Hereditary angioedema (HAE) is a rare disease caused by the decrease or functional defect of C1 lipase inhibitor in the serum of patients, resulting in excessive activation of C1, uncontrolled cleavage of C4 and C2, and increased production of complement kinin.
Increased microvascular permeability
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Most cases of hereditary angioedema result from decreased levels (type I) or decreased function (type II) of C1 esterase inhibitors
Vascular antisense oligonucleotide Donidalorsen is a potential "best-in-class" prophylactic therapy for hereditary angioedema that reduces HAE frequency and disease burden by selectively inhibiting prekallikrein .
In a recent phase II clinical study, patients with hereditary angioedema with C1 deficiency suppression were treated with Donidalsen (80 mg) or placebo 4 times every 4 weeks
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The primary endpoint was the rate of angioedema episodes from week 1 (baseline) to week 17
Quality of Life
Twenty patients participated in the study, 14 received Donidalorsen and 6 received placebo
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In patients treated with Donidalorsen, the mean monthly incidence of confirmed angioedema was 0.
23 versus 2.
In patients treated with Donidalorsen, the mean monthly incidence of confirmed angioedema was 0.
Significant reduction in risk of edema episodes following Donidalorsen treatment
Significantly lower risk of edema episodes after Donidalorsen treatmentIn this small phase II trial of patients with hereditary angioedema, treatment with Donidalorsen significantly reduced the rate of angioedema episodes and improved quality of life
.
.
In this small phase II trial of patients with hereditary angioedema, treatment with Donidalorsen significantly reduced the rate of angioedema episodes and improved quality of life
Original source:
Lauré M.
Inhibition of Prekallikrein for Hereditary Angioedema.
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