Hereditary angioedema (HAE) is a human genetic disorder caused by a shortage of C1 inhibitor activity. The disease is characterized by acute attacks of painful swelling of soft tissues (edema), including regions of the skin, the intestine, and the mouth and throat. If the soft tissue of the throat is involved, an attack of angioedema can be fatal.
Pharming’s marketing authorization application (MAA) is supported by clinical and pharmaceutical data on recombinant human C1 inhibitor (rhC1INH) as required by the European Medicines Agency (EMEA). In clinical studies, all HAE patients treated with rhC1INH demonstrated a rapid time to beginning of relief and time to minimal symptoms.
The rhC1INH product has already received orphan medicinal product designation for HAE in Europe, which provides 10 years of market exclusivity for an approved recombinant human C1 inhibitor product.
Pharming’s rhC1INH could represent the first new therapy for HAE patients in over 30 years if approved. The only approved product for the treatment of HAE attacks is plasma derived C1 inhibitor, which is available in a limited number of European countries.