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.
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
It's hard to live this way. It’s hard to live with HAE without being on treatment.
OVERVIEW OF HEREDITARY ANGIOEDEMA (HAE)
- 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
- 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.
Common locations of swelling8
The World Allergy Organization (WAO) guidelines recommend the use of C1-INH for HAE for long-term prophylaxis.9
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
FOR YOUR PATIENTS
To me, any therapy that replaces what you're actually missing and keeps it at a constant level, makes sense.
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).
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."