Pharming submits supplemental BLA to FDA for Ruconest for prophylaxis of hereditary angioedema attacks

The submission includes data from two completed trials of RUCONEST® for the prophylaxis of HAE attacks: a randomized, double-blind, placebo-controlled trial and an open-label study. The two studies enrolled a total of 56 patients and showed consistent efficacy and safety results.

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.

Pharming is a specialty pharmaceutical company developing innovative products for the safe, effective treatment of rare diseases and unmet medical needs. Pharming's lead product, RUCONEST® (conestat alfa) is a recombinant human C1 esterase inhibitor approved for the treatment of acute Hereditary Angioedema ("HAE") attacks in patients in Europe, the US, Israel and South Korea.

The product is available on a named-patient basis in other territories where it has not yet obtained marketing authorization.