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Ruconest Gets FDA Nod for Treatment of Hereditary Angioedema
The FDA has approved Ruconest (C1 esterase inhibitor [recombinant], Salix Pharmaceuticals/Pharming Group) 50 IU/kg for the treatment of acute angioedema attacks in adult and adolescent patients with hereditary angioedema (HAE). Because of the limited number of patients with laryngeal attacks, the effectiveness of Ruconest in this setting was not established.
Ruconest is a recombinant C1 esterase inhibitor that can be administered by the patient after receiving training by a health care provider.
HAE attacks are caused by a deficiency of the C1 inhibitor protein in the blood. HAE is a rare inherited genetic condition that is often not properly diagnosed until later in a patient’s life as the symptoms of an attack can mirror an allergic reaction. Severe, painful swelling can occur at any time.
The FDA’s approval of the biologics license application (BLA) for Ruconest for the treatment of acute angioedema attacks in patients with HAE was based on the results of a phase III, randomized, double-blind, placebo-controlled trial (RCT) that included an open-label extension (OLE) phase. This study was supported by the results from two additional RCTs and two additional OLE studies.
The pivotal RCT and OLE trials evaluated a total of 44 subjects who experienced 170 HAE attacks. The trials’ primary efficacy endpoint was the time to the beginning of symptom relief, which was assessed using patient-reported responses to two questions about the change in overall severity of their HAE attack symptoms after the start of treatment. These symptoms were assessed at regular time points for each of the affected anatomical locations for up to 24 hours. To achieve the primary endpoint, a patient had to have a positive response to both questions along with persistent improvement at the next assessment time (i.e., the same or better response).
A statistically significant difference in the time to the beginning of symptom relief was observed in the intent-to-treat population (n = 75) for Ruconest compared with placebo (P = 0.031); the median time to the beginning of symptom relief was 90 minutes for Ruconest (n = 44) and 152 minutes for placebo (n = 31).
Source: Salix Pharmaceuticals; July 17, 2014.