MANAGE C1-INH DEFICIENCY WITH HAEGARDA

HAEGARDA replaces missing or dysfunctional C1-INH

Functional C1‑INH levels ≥40% of normal are proposed to reduce the risk of HAE attacks¹
HAEGARDA replaces missing or dysfunctional C1-INH, which regulates the normal production of bradykinin

What made me try HAEGARDA? It’s the same protein my body is lacking.

-Zahra, HAEGARDA Advocate

image showing increase c1-ing levels and decreased risk for attacks

Subcutaneous C1-INH builds and maintains steady-state C1-INH functional activity²*

Administered subcutaneously, HAEGARDA 60 IU/kg maintained steady-state C1-INH functional levels above 40%.²
Steady state is expected within 3 to 4 doses²

* The plasma levels of C1-INH functional activity were evaluated in patients with type 1 or type 2 HAE in a Phase 3, placebo-controlled, crossover study. Patients received twice-weekly subcutaneous injections of HAEGARDA 40 IU/kg or 60 IU/kg for 16 weeks. The model-derived outcome is the steady-state C1-INH functional activity vs time.²

ROOT CAUSE OF HAE:
DEFICIENT OR DYSFUNCTIONAL C1-INH³

Missing or nonfunctioning C1-INH is the root cause of HAE⁴

C1-INH inactivates factors Xlla, plasmin, and kallikrein, thus preventing bradykinin overproduction⁵
Low-level of dysfunctional C1-INH allows bradykinin production to go unregulated⁶
Excess bradykinin increases vascular permeability, resulting in angioedema⁴
HAE with normal C1-INH levels and function is very rare; lack of pivotal clinical trials hinders ability to clearly establish diagnosis⁷

OVERVIEW OF HEREDITARY ANGIOEDEMA (HAE)

Epidemiology

HAE is rare: affecting ~1 in 50K individuals. (1 in 10K – 1 in 150K worldwide)⁸
HAE is an autosomal dominant genetic disorder resulting in deficiency (type I-85%) or dysfunction (type II-15%) of the C1-INH protein⁸
C1-INH—a serine protease inhibitor—regulates the complement, contact, and coagulation cascades⁸
Deficiency of functional C1-INH allows for uncontrolled activation of the contact system, resulting in increased vascular permeability and the classic symptoms of swelling⁸

Age of onset may vary

Symptoms may occur at any age, but initial onset usually begins during childhood or adolescence (median = 6 years of age). Fifty percent of female and male patients are symptomatic at 12 and 13 years of age, respectively⁹
HAEGARDA is now approved for adults and children 6 years and older

Symptoms

Unpredictable angioedema attacks of swelling in various body parts¹⁰
Debilitation due to extreme pain, vomiting, nausea, and, if the airway is affected, asphyxiation¹⁰
Patients may experience symptoms for 10 or more years before HAE is identified¹¹

I remember the first time I went into the emergency room and the doctor said, ‘You have HAE’ and…it was such a load removed from my shoulders because it was a doctor who knew what I had, and was able to treat it.

-Leah, HAEGARDA Advocate

Common locations of swelling⁸

The World Allergy Organization (WAO) guidelines recommend the use of C1-INH for HAE for long-term prophylaxis.⁹

The WAO Guideline for the Management of HAE states that patients should have HAE rescue medication available at all times.⁹

What Makes HAEGARDA Different

HAEGARDA is the HAE product that reduced HAE attacks by 95%.^†

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^† Median reduction in the number of attacks with HAEGARDA 60 IU/kg vs placebo.

THE WORLD ALLERGY ORGANIZATION GUIDELINES RECOMMENDATIONS⁹

C1-INH has been used for 35 years, and is the WAO-recommended first-line therapy

The WAO recommends C1-INH as first-line preventive therapy for HAE for long-term prophylaxis⁴
C1-INH is the preferred therapy for pregnant or breastfeeding HAE patients

SEE THE SUPPORT AVAILABLE
FOR YOUR PATIENTS

To me, any therapy that replaces what you're actually missing and keeps it at a constant level, makes sense.

-Leah, HAEGARDA Advocate

HAE TREATMENT GOALS FOR HCPs⁹

On-demand treatment goal^‡

Minimize the duration and severity of an attack that has started
HAEGARDA is not approved for on-demand use

^‡ Short-term or preprocedural prophylaxis can also be administered before an event that may trigger an HAE attack (eg, medical or invasive dental procedure).

See On-demand Treatment of Acute HAE Attacks

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Long-term prophylaxis (LTP) treatment goals

Prevent attacks and reduce severity if they occur
Minimize the patient's burden of disease

World Allergy Organization/European Academy of Allergy and Clinical Immunology
guidelines on prophylaxis
"We recommend that patients are evaluated for long-term prophylaxis at every visit. Disease burden and patient preference should be taken into consideration. Evidence Grade D; strength of recommendation strong, 100% agreement."

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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. Zuraw BL, Cicardi M, Longhurst HJ, et al. Phase II study results of a replacement therapy for hereditary angioedema with subcutaneous C1-inhibitor concentrate. Allergy. 2015;70(10):1319-1328. 3. Weiler CR,van Dellen RG. Genetic test indications and interpretations in patients with hereditary angioedema. Mayo Clinic Proc. 2006;81(7):958-972. 4. Zuraw BL. The pathophysiology of hereditary angioedema. World Allergy Organ J.2010;3(9 Suppl):S25-S28.1 5. Kaplan AP et al. Clinic Rev Allerg Immunol. 2016;51(2):207-215. 6. Kaplan AP. Angioedema. World Allergy Organ J. 2008;1(6):103-113. 7. Zuraw BL. Hereditary angioedema with normal C1 inhibitor:Four types and counting. J Allergy Clin Immunol. 2018; 141(3):884-885. 8. Lumry WR. Overview of Epidemiology, Pathophysiology, and Disease Progression in Hereditary Angioedema. Am J Manag Care. 2013;19:S103-S110. 9. Maurer M et al. The international WAO/EAACI guideline for the management of hereditary angioedema– the 2017 revision and update. World Allergy Organ J. 2018;11:5. 10. Zuraw BL. Hereditary angioedema. N Engl J Med. 2008;359(10):1027-1036. 11. MacGinnitie AJ. Pediatric hereditary angioedema. Pediatr Allergy lmmunol. 2014;25(5):420-427. 12. Craig T, Zuraw B, Longhurst H, et al. Long-term outcomes with subcutaneous C1-inhibitor replacement therapy for prevention of hereditary angioedema attacks. J Allergy Clin Immunol Pract. 2019 Feb 15. DOI: https://doi.org/10.1016/j.jaip.2019.01.054.