Ruconest, an injectable human recombinant C1-esterase inhibitor, has been approved by the Food and Drug Administration for treating acute angioedema attacks in adults and adolescents with hereditary angioedema.
Ruconest is the first recombinant C1-esterase inhibitor to be approved by the agency, according to the July 17 FDA statement announcing the approval. Ruconest "is intended to restore the level of functional C1-esterase inhibitor in a patient’s plasma, thereby treating the acute attack of swelling" in patients with hereditary angioedema (HAE). HAE is caused by an insufficient amount of C1-esterase inhibitor, a plasma protein, the statement said. HAE symptoms include acute attacks of swelling of the hands, feet, limbs, face, intestinal tract, or airway, which can occur spontaneously or are triggered by stress, surgery, or an infection, according to the FDA.
Ruconest is expected to be available "later in 2014," according to the press release on the approval issued by Salix Pharmaceuticals.
Santarus, a subsidiary of Salix, will distribute the drug in the United States; the drug is manufactured by the Pharming Group NV, in the Netherlands.
A Salix press release issued on July 17 provided details of the study that was the basis of the approval, a randomized, double-blind, placebo-controlled phase III trial, which included an open-label extension phase, of adults and adolescents who had 170 HAE attacks. The time to the beginning of symptom relief, based on patient reported responses, was significantly faster among those treated with Ruconest, administered at the time of the attack, compared with those on placebo – a median of 90 minutes among the 44 patients treated with Ruconest vs. a median of 152 minutes among the 31 patients who received a placebo, a statistically significant difference, according to Salix.
However, "Because of the limited number of patients with laryngeal attacks, effectiveness was not established in HAE patients with laryngeal attacks," according to the Salix statement.
Headache, nausea, and diarrhea were the most common adverse events associated with treatment, the FDA statement said.
Ruconest was designated an orphan drug because it is intended to treat a rare disease or condition; about 6,000-10,000 people in the United States have hereditary angioedema, according to the FDA.