Berotralstat for preventing recurrent attacks of hereditary angioedema in children aged 2 to <12 years

Berotralstat is in development for the routine prevention of recurrent attacks of hereditary angioedema (HAE) in children aged 2 to <12 years. HAE is a rare disease, caused by a gene alteration that means an important protein in the immune system, C1 esterase inhibitor (C1-INH), is missing or dysfunctional in these patients. Lack of the C1-INH protein triggers the body’s immune system to misfire through a series of downstream signalling pathways that cause excessive production of a protein called bradykinin. Bradykinin acts on blood vessels making them wider and more permeable (leaky) to fluid. Fluid that has leaked from the blood vessels accumulates into surrounding tissues causing rapid swelling (oedema). There are currently no NICE recommended treatments options for preventing recurrent attacks of HAE in children aged under 12 years.