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Hereditary angioedema (HAE) is a genetic disorder that falls under the purview of allergy and immunology, characterized by recurrent episodes of severe swelling (angioedema). These episodes can affect various parts of the body, including the face, extremities, gastrointestinal tract, and airway, potentially resulting in life-threatening situations such as asphyxiation if the airway is obstructed. The condition is caused by a deficiency or dysfunction of the C1 inhibitor protein, which is involved in regulating inflammatory pathways. Long-term management includes prophylactic treatments to prevent attacks and acute therapies aimed at treating swelling episodes as they occur. The HAE market includes drugs that replace the deficient C1 inhibitor, synthetic peptides that inhibit the kinin-generating pathways, and monoclonal antibodies that target and negate proteins involved in the swelling process. The approach to treatment is individualized, considering the severity of the condition, frequency of attacks, and patient-specific factors.
Within the hereditary angioedema market, several pharmaceutical companies are active participants. Key players include Shire (now part of Takeda Pharmaceutical Company), CSL Behring, and Pharming Group N.V. These companies offer FDA-approved treatments for HAE, including replacement therapies and prophylactic drugs. Additionally, BioCryst Pharmaceuticals provides an orally administered therapy, expanding the options for patients who prefer not to use injectables. Other emergent companies continue to invest in research and development aimed at improving therapeutic outcomes and patient Show Less Read more