THE RIGHT THERAPY MATTERS FOR ATTACK-FREE DAYS

HAEGARDA IS THE HAE THERAPY THAT REDUCED ATTACKS BY 95%^*

COMPACT PHASE 3 STUDY^1†

PRIMARY ENDPOINT

Chart showing 95% overall reduction in HAE attacks per month relative to placebo

^* Median reduction in number of attacks in patients receiving 60 IU/kg of HAEGARDA vs placebo.

^† Clinical study for Optimal Management of Preventing Angioedema with low-volume subcutaneous C1-inhibitor replacement Therapy.

C1-INH=C1 inhibitor.
HAE=hereditary angioedema.

See COMPACT Phase 3
Study design

SECONDARY ENDPOINT

The WAO Guideline for the Management of HAE states that patients should have HAE rescue medication available at all times.²

^‡ Median reduction in rescue medication use in patients receiving 60 IU/kg of HAEGARDA vs placebo.

EXPLORATORY ENDPOINT

Severity of HAE Attacks¹

9% of patients on HAEGARDA 60 IU/kg experienced a severe attack, compared to 69% on placebo¹
Attacks were graded by investigators on the intensity of the most severe symptom

Living without HAE attacks allows me to be more independent, confident, and free.

- Zahra, HAEGARDA Advocate

HAEGARDA PROVIDES ATTACK‑FREE DAYS FOR THE LONG TERM

COMPACT OPEN-LABEL EXTENSION STUDY—SUBGROUP ANALYSIS

IN A POST-HOC ANALYSIS OF PATIENTS TREATED WITH HAEGARDA

This post-hoc analysis is the longest reported use of HAEGARDA to date³

^§In a subgroup analysis of 24 U.S. patients receiving 60 IU/kg of HAEGARDA eligible to continue the open-label extension study for >12 months.³

See open-label extension
study design

NEXT: C1-INH DEFICIENCY

References: 1. Longhurst H, Cicardi M, Craig T, et al. Prevention of hereditary angioedema attacks with a subcutaneous Cl inhibitor. N Engl J Med. 2017;376(12):1131-1140. 2. Maurer M, Magerl M, Betschel S, et al. The international WAO/EAACI guideline for the management of hereditary angioedema—the 2021 revision and update. Allergy. 2022;77(7):1961-1990. 3. Craig T, Feuersenger H, Pragst I, et al. Prophylactic therapy with subcutaneous C1-inhibitor is associated with sustained symptom control in patients with hereditary angioedema. Allergy Asthma Proc. 2022;43:202-208.

HAEGARDA PIVOTAL STUDY DESIGN

The COMPACT^* Phase 3 Study aimed to evaluate the efficacy and safety of HAEGARDA in reducing the frequency of hereditary angioedema (HAE) attacks¹

32-week, randomized, double-blind, placebo-controlled, crossover study

90 patients including adults and adolescents (12 to 72 years of age) with symptomatic type 1 or type 2 HAE

HAEGARDA 60 IU/kg, HAEGARDA 40 IU/kg, or placebo was administered twice weekly (every 3 or 4 days)

Baseline HAE attack frequency was a mean of 8.8 in the 3 months before screening for the 60-IU/kg group

* Clinical study for Optimal Management of Preventing Angioedema with low-volume subcutaneous C1-inhibitor replacement Therapy.

Reference: 1. Longhurst H, Cicardi M, Craig T, et al. Prevention of hereditary angioedema attacks with a subcutaneous Cl inhibitor. N Engl J Med. 2017;376(12):1131-1140.