Pharming submits supplemental Biologics License Application to FDA for RUCONEST® for prophylaxis of hereditary angioedema attacksMonday, November 27, 2017
Dr. Bruno Giannetti, MD PhD, Chief Operations Officer of Pharming, commented: “HAE patients in the US are currently facing a shortage of plasma-derived C1 inhibitor used to prevent attacks. We understand that this supply disruption has had serious consequences for them, including additional stress, disease-related complications, and hospitalizations. We look forward to working with FDA and potentially providing these patients an alternative and plasma free option for HAE prophylaxis.”
Hereditary Angioedema (“HAE”) is a rare genetic disorder. It is characterized by spontaneous and recurrent episodes of swelling (edema attacks) of the skin in different parts of the body, as well as in the airways and internal organs. Edema of the skin usually affects the extremities, the face, and the genitals. Patients suffering from this kind of edema often withdraw from their social lives because of the disfiguration, discomfort and pain these symptoms may cause. Almost all HAE patients suffer from bouts of severe abdominal pain, nausea, vomiting and diarrhoea caused by swelling of the intestinal wall.
Edema of the throat, nose or tongue can be particularly dangerous as this can lead to obstruction of the airway passages and be potentially life threatening. Although there is currently no known cure for HAE, it is possible to treat the symptoms associated with edema attacks. HAE affects about 1 in 10,000 to 1 in 50,000 people, worldwide experts believe that a lot of patients are still seeking the right diagnosis: although HAE is (in principle) easy to diagnose, it is frequently identified very late or not discovered at all. The reason HAE is often misdiagnosed is because the symptoms are similar to those of many other common conditions such as allergies or appendicitis by the time it is diagnosed correctly, the patient has often been through a long-lasting ordeal.