The definition describes HAE as a genetic disease. No genes, OMIM entries, inheritance patterns or molecular mechanism are populated in this packet, so the specific genetic causes and mode of inheritance are not described here. Listed subtypes include forms with C1 inhibitor deficiency and forms with normal C1 inhibitor, but the packet does not detail them.

The packet does not certify diagnostic methods, criteria or newborn screening information for hereditary angioedema.

The packet lists four FDA-approved treatments for hereditary angioedema with an active market status: berotralstat (Orladeyo, approved 2025-12-11), donidalorsen (Dawnzera, 2025-08-21), sebetralstat (Ekterly, 2025-07-03) and garadacimab (Andembry, 2025-06-16). Additional orphan drug designations are listed for other products, including a gene-editing product and a gene transfer vector. Designation is not approval, and these are not described as approved.

Natural history and long-term outcome data are not certified in this packet. Survival and individual outcomes are not described.

The packet lists ClinicalTrials.gov records for hereditary angioedema, including several involving newer approaches. Intellia Therapeutics sponsors NTLA-2002 studies: a Phase 3 study (HAELO, NCT06634420) and a Phase 1 study in adults (NCT05120830), both active and not recruiting. ADARx Pharmaceuticals sponsors the Phase 3 STOP-HAE study of ADX-324 (NCT06960213, recruiting), and Ionis a Phase 3 study of donidalorsen in children (NCT07298447, recruiting). Listing of a trial does not indicate that an intervention is effective.