FDA OKs Recombinant C1-Inhibitor (Ruconest) for Hereditary Angioedema

Megan Brooks

Disclosures

July 17, 2014

The US Food and Drug Administration (FDA) has approved the first and only plasma-free recombinant C1-esterase inhibitor (Ruconest, Pharming Group and Salix Pharmaceuticals) for the treatment of acute attacks of hereditary angioedema (HAE) in adolescents and adults.

HAE is a rare and potentially life-threatening genetic disorder linked to a defect in the C1-esterase inhibitor protein. The disorder is characterized by relapsing, self-limiting episodes of edema that occur primarily in the extremities, intestinal tract, face, and larynx. Swelling of the airway is potentially fatal without immediate treatment.

Ruconest 50 IU/kg is intended to restore the level of functional C1-esterase inhibitor in plasma, thereby treating the acute attack of swelling, the FDA notes in a statement issued July 17, announcing the approval.

Ruconest is "an important treatment option for these patients," Karen Midthun, MD, director of the FDA's Center for Biologics Evaluation and Research, said in the statement.

The safety and efficacy of Ruconest was demonstrated in a multicenter controlled trial involving 44 adult and adolescent patients who experienced 170 HAE attacks.

Because of the limited number of patients with laryngeal attacks, effectiveness was not established in patients with HAE with laryngeal attacks, the company notes.

Ruconest "provides the HAE community with another FDA-approved option for treating painful and debilitating HAE attacks," Anthony Castaldo, president of the nonprofit Hereditary Angioedema Association, said in a Salix news release.

The most common adverse reactions (incidence ≥2%) reported in patients treated with Ruconest were headache, nausea, and diarrhea.

Ruconest is contraindicated in patients with a history of allergy to rabbits or rabbit-derived products and patients with a history of life-threatening immediate hypersensitivity reactions to C1-esterase inhibitor preparations.

The company says serious arterial and venous thromboembolic (TE) events have been reported at the recommended dose of plasma-derived C1-esterase inhibitor products in patients with risk factors, which may include the presence of an indwelling venous catheter/access device, prior history of thrombosis, underlying atherosclerosis, use of oral contraceptives or certain androgens, morbid obesity, and immobility. Patients with known risk factors for TE events should be monitored during and after administration of Ruconest, the company says.

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