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27 July 2014

Ruconest for Treatment of Hereditary Angioedema (HAE)

Ruconest (C1 Esterase Inhibitor [Recombinant]) is a recombinant approved for treatment of acute angioedema attacks in patients suffering from hereditary angioedema (HAE).
Glybera is the first gene therapy approved in Europe.
The European Commission’s approval of Eliquis was based on ARISTOTLE and AVERROES clinical studies. Image courtesy of Bristol-Myers Squibb.
Synribo can be subcutaneously injected.

Ruconest hae treatment

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Ruconest (C1 esterase inhibitor [recombinant]) is a recombinant approved for the treatment of acute angioedema attacks in patients suffering from hereditary angioedema (HAE). The drug is manufactured by Pharming Group and marketed by Salix Pharmaceuticals in the US.

Ruconest was approved by the US Food Drug and Administration (FDA) in July 2014 for treatment of acute angioedema attacks in adult and adolescent patients with HAE. The FDA has also granted orphan drug designation to Ruconest and seven years of marketing exclusivity to Salix Pharmaceuticals in the US.

Hereditary angioedema (HAE) causes and symptoms



A drug developed by US-based Dyax Corp for the treatment of hereditary angioedema (HAE).


HAE is a genetic disorder resulting from deficiency of C1 esterase inhibitor (C1-INH) protein, which has a normal function of regulating several inflammatory pathways in the body. Shortage of the protein leads to the leakage of fluids from blood vessels into connective tissue. It further leads to recurrent attacks of angioedema (local swelling), often associated with swelling and severe pain. When left untreated, these symptoms may become life-threatening.

HAE patients experience swelling in their face, abdomen and extremities. Abdominal swelling leads to nausea, vomiting and severe pain. Swelling in other parts is often painful, disabling and disfiguring. HAE is a very rare disorder that affects one in 10,000 to 15,000 people.

Ruconest’s mechanism of action

Ruconest is the first FDA-approved recombinant C1 esterase inhibitor for the treatment of HAE. The drug has to be administered at the onset of angioedema attack symptoms. It brings the patient’s C1-INH levels to the normal range, relieving from the symptoms. The drug is available in the form of 50IU/kg injection.

Clinical trials on Ruconest

"Results demonstrating Ruconest to be a safe and effective option to treat patients suffering from acute hereditary angioedema attacks."

Pharming conducted two Phase III clinical trials on the recombinant human C1 inhibitor (rhC1INH) to evaluate the safety and efficacy in treatment of acute attacks of HAE. Studies enrolled 75 HAE patients, with results demonstrating Ruconest to be a safe and effective option to treat patients suffering from acute hereditary angioedema attacks.

The open label, randomised, placebo-controlled, double blind study analysed the results from 44 subjects who experienced 170 HAE attacks. The study evaluated the safety and efficacy of Ruconest in two treatment arms. Subjects in the double-blind phase one were randomised to receive 100IU/kg of rhC1INH or Saline solution in 1:1 ratio once.

The study’s primary outcome measure was the time of beginning of the relief symptoms once the drug is administered, which was assessed with a patient-reported visual analogue scale (VAS) ranging from 0mm (no symptoms) to 100mm (extremely disabling). At the first location, the first VAS score was recorded as 20mm from the baseline score (t=0 min).

The secondary outcome measure was the time to minimal symptoms for an attack, which was also assessed using VAS score. Symptoms were minimal at all the locations at a VAS score below 20mm.

A significant difference in the time to beginning of symptoms was observed in the patients treated with Ruconest, when compared with the scores received from placebo arm. The average time was 90 minutes for Ruconest, whereas it was 153 minutes for the placebo arm.

The most common adverse effects recorded were nausea, diarrhoea and headache. One severe adverse reaction recorded in Ruconest group was anaphylaxis.

Marketing commentary

Pharming Group manufactures Ruconest while Salix Company has purchased the rights from Pharming to commercialise the product in North America. The drug is planned to be released in the market in the second half of 2014.

Ruconest is also approved in European Union (EU) and marketed by Swedish Orphan Biovitrum in the EU.

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