C1 Esterase Inhibitor (Ruconest) Shows Promise in Treating Recurrent Angioedema
FDA review set for April 2014 (November 8)
New data from an open-label extension of a pivotal phase III clinical trial of Ruconest (recombinant human C1 esterase inhibitor [rhC1INH], Santarus, Inc.) will be presented Nov. 9 and 10 at the 2013 American College of Allergy, Asthma, and Immunology Annual Scientific Meeting in Baltimore, Maryland.
Ruconest is an investigational drug in the U.S. and has been granted an orphan drug designation by the FDA for the treatment of acute attacks of hereditary angioedema (HAE). The Biologics License Application (BLA) filing for Ruconest is under review, and a response is expected by April 16, 2014.
In the open-label extension trial, Ruconest was administered for the treatment of 224 repeated acute angioedema attacks in 44 patients with HAE after initial treatment.
The median time to the onset of symptom relief after treatment, as measured by patient responses to a Treatment Effect Questionnaire (TEQ), for the first five repeated attacks ranged from 62.5 to 134.0 minutes, and was 75.0 minutes across all attacks. The median time to minimal symptoms (the first three attacks per patient), as measured by a TEQ, ranged from 243 to 304 minutes, and was 303 minutes for all assessed attacks.
Other key findings include the following:
- Only one dose of Ruconest was administered for 96% of the 224 attacks.
- Twelve of 44 patients (27%) experienced at least one treatment-emergent adverse event (TEAE) within 72 hours of completing the Ruconest infusion.
- TEAEs occurring in 5% of patients were nasopharyngitis, cough, increased fibrin D-dimer, and headache.
- The percentage of patients experiencing TEAEs did not increase with Ruconest treatments for recurrent attacks.
- There were no discontinuations because of adverse events, no thrombotic or anaphylactic events, and no neutralizing anti-C1INH antibodies observed with repeated Ruconest treatment.
Source: Santarus, Inc.; November 8, 2013.