new in 2023 !
What is HAE?
- Hereditary Angioedema (HAE) is a rare hereditary disease that can cause attacks of swelling, and often pain, in specific parts of the body including the stomach, hands, feet, arms, legs, genitals, throat, and face. Depending on the severity of the disease, some people have many attacks each month, while others go months without an attack.
- People with HAE are missing, or have low levels of, the protein C1 esterase inhibitor (C1-INH) which is a component of the complement pathway; in some cases, the C1-INH levels are sufficient, but the protein does not function properly.
- The current understanding of the pathophysiology of HAE has now expanded beyond the complement pathway and can include genetic mutations in the SERPING1, F12, PLG, and ANGPT1 genes. Plasma kallikrein inhibitors have been developed to inhibit the kallikrein-kinin pathway to prevent (prophylactic) and treat on-demand (acute) HAE attacks.
Types of HAE
- Hereditary Angioedema is classified into three types based on what problem the genetic defect causes.
- Type I - Low levels of C1-INH in the body; this is the most common form of HAE.
- Type II - Normal levels of C1-INH, but it does not function normally; this accounts for ~15% of people with HAE.
- Type III - Normal levels of C1-INH and it functions normally; this is extremely rare and not well understood.
HAE treatment options
- Until recently, options for addressing HAE were limited to on-demand treatments administered either via IV or subcutaneously.
- The recent introduction of berotralstat (marketed under the brand name Orladeyo), a once-daily, orally-administered preventative treatment has disrupted the market, but is likely to be only the first of several new treatments for HAE that will soon be available.
What is HAEsyndiTrack?
- APLUSA is launching a new syndicated Real World Data (RWD) Market Research to propose market surveillance in this challenging disease with new emerging options.
- The introduction of subcutaneous and oral treatments for hereditary angioedema (HAE) has been a boon to patients and disruptive to the marketplace. With many more SC and oral treatments in mid- to late-stage development, the HAE market is about to become extremely competitive.
- It is with this dynamic landscape in mind that APLUSA launched HAEsyndiTrack.
- This study will feature:
• Real-world treatment data from patient charts
• Prescriber attitudes, awareness, and treatment patterns for current
• Patient attitudes and awareness of current
• Assessment of field force and messaging effectiveness
• Future market trends and likely prescribing shifts
Inclusion / Exclusion screening criteria
- Physicians must manage at least 2 adult patients with Type I or Type II HAE
- Patient cases recorded
- Patients must be 18+ and diagnosed with HAE Type I or II and have received treatment in the past 6 months
- On average: 3 PRFs per physician per wave
- 750 PRFs across the G7
Fielding of HAEsyndiTrack
- The inaugural wave of the study will field
- in the US (100 Immunologists / Allergists + 300 patient record forms) in July,
- with the remaining G7 countries - France, Germany, Italy, Spain, UK, and Japan - to follow.
- (Additional countries are available upon request).
- The actual timeline and availability of results will be discussed and negotiated with all founding clients.
Data captured by HAEsyndiTrack
- The study will capture insights on pipeline and inline products for both on-demand and prophylactic usage, including:
- Firazyr (icatibant injection)
- Berinert (C1 esterase inhibitor, human)
- Ruconest (C1 esterase inhibitor, recombinant)
- Cinryze (C1 esterase inhibitor, human)
- Kalbitor (ecallantide)
- Haegarda (C1 esterase inhibitor, human, subcutaneous)
- Takhzyro (lanadelumab-flyo, injection)
- Orladeyo (berotralstat)
- deucrictibant
- donidalorsen
- garidacimab
- KVD824
- BCX4161