what is Hereditary Angioedema (HAE)?
Hereditary angioedema (HAE) is a rare and potentially life-threatening genetic disease that occurs in approximately 1 in 10,000 to 1 in 50,000 people, according to HAEi.
ultra rare
1 in 10,000 – 50,000*
- USA 7,000-15,000* people
- EU 9,000-15,000* people
- Japan 2,500-4,500* people
genetic
Enzyme defect
- Decreased production or production of functionally impared C1 esterase inhibitor (C1-IHN)
hereditary
Inherited
- Generally passed on from parents to children
how are those with HAE affected?
People living with HAE experience unpredictable and prolonged attacks of edema (swelling), which can occur in the upper and lower limbs, face, gastrointestinal tract, genitals, and airway resulting in severe swelling and pain, nausea, diarrhea, and vomiting, and may be life-threatening in case of airway blockage. Depending on the severity of the disease, some people will have many attacks each month, while others may go months without an attack.
unpredictable swelling attacks
- Extremely painful
- Limiting daily activity
- Stress related
variable degree of severity
- Frequency from 1 per year to multiple per week
- Severity: mild to severe
different locations
- Facial area
- Extremities
- Abdomen
treatment of HAE
Treatment of HAE consists of long-term prophylaxis, management of acute attacks, and intermittent episodic prophylaxis in advance of known triggers.
currently, those with HAE are forced to choose between effectiveness and ease of therapy.
people with HAE deserve to have it all!
A first-in-class, oral small-molecule antagonist of the bradykinin B2 receptor may provide an effective and more convenient way to manage HAE and improve quality of life. Pharvaris aims to bring better oral options to meet the needs of people with HAE for on-demand and prophylactic treatment.
PHVS416 and PHVS719 are designed to directly target the ultimate event in the HAE cascade, the interaction of bradykinin with the bradykinin 2 receptor, a proven mechanism for the treatment of HAE attacks. These oral small molecules are designed to have a quicker onset, higher potency, and longer half-life.
Multiple therapies are available, acting at different points in the bradykinin-forming cascade. Only one approved therapy, icatibant, acts directly at the bradykinin B2 receptor to treat all types of HAE by inhibiting bradykinin interaction with its bradykinin B2 receptor target, and hence by putting a halt to bradykinin-mediated signaling.
All current therapies, including icatibant, are limited by invasive routes of drug administration (injection or infusion), inconvenient dosing regimens, or undesired side effects. Surveys of people living with HAE overwhelmingly show that they seek additional treatment options.
