MAKE HAEGARDA YOUR FIRST-LINE PROPHYLACTIC HAE THERAPY OF CHOICE.
HAEGARDA IS THE ONLY PREVENTIVE THERAPY THAT REDUCES HAE ATTACKS BY 95%*
Secondary Endpoint
Number of Uses of Rescue Medication Per Month Relative to Placebo1†

† Median reduction in rescue medication use with HAEGARDA 60 IU/kg vs placebo.
The WAO Guideline for the Management of HAE states that patients should have HAE rescue medication available at all times.2
Exploratory Endpoint
Severity of HAE Attacks1

- Only 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.
LONG-TERM USE OF SUBCUTANEOUS C1-INH PROVIDES A SAFE AND SUSTAINED PROPHYLACTIC THERAPEUTIC EFFECT3
The COMPACT‡ open-label extension study investigated the long-term safety of HAEGARDA.
Study Objectives
- The primary objective was to determine the long-term safety of subcutaneous C1-INH (SC)
- Secondary endpoints assessed additional safety parameters and efficacy of subcutaneous C1-INH (SC)
Study Results
- Average duration of exposure was 1.4 years; 41 patients (34%) had exposure for >2 years
- The patients studied reported a mean of 4.3 attacks per month in the 3 months prior to study entry
- A post-hoc analysis showed the median time to first attack in the study was 224 days with HAEGARDA 60 IU/kg (n=63)4

PER YEAR COULD BE A REALITY
FOR YOUR PATIENTS
In a long-term study following patients over 1.4 years, HAEGARDA reduced the HAE attack rate to 1 per year.3§
§ Median attack rate in patients who were followed an average of 1.4 years.
Study Conclusion
- Long-term subcutaneous C1-INH replacement therapy provides a safe and sustained therapeutic effect
study design
‡ Clinical study for Optimal Management of Preventing Angioedema with low-volume subcutaneous C1-inhibitor replacement Therapy.
C1-INH is the World Allergy Organization recommended first-line, long-term prophylaxis therapy.
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.
COMPACT* OPEN-LABEL EXTENSION STUDY
First and largest long-term study to investigate the safety and prophylactic effects of twice-weekly subcutaneous C1-INH over an extended period in patients with frequent HAE attacks.

- Per the study design, subjects from the United States who completed the 1-year study period of the open-label extension study were eligible to continue in the study for an additional 88 weeks
C1-INH=C1 inhibitor.
HAE=hereditary angioedema.
* Clinical study for Optimal Management of Preventing Angioedema with low-volume subcutaneous C1-inhibitor replacement Therapy.
† Only the 60-IU/kg dose of HAEGARDA is approved for use.