Xolair Shows Promise for Idiopathic Angioedema: Phase 4 Trial
Medication for chronic hives seen to lessen angioedema activity, boost life quality
Xolair (omalizumab), an approved therapy for allergic asthma and chronic spontaneous urticaria (CSU), shows promise for treating idiopathic angioedema, according to data from a small Phase 4 trial.
In the study, treatment for roughly six months with Xolair as an add-on therapy lessened disease activity when compared to a placebo. Signs of remission of disease activity were seen in four of the five patients who received Xolair.
Trial data was reported in a study, “Omalizumab for treatment of idiopathic angioedema,” published in the journal Annals of Allergy, Asthma & Immunology.
CSU, also known as chronic idiopathic urticaria, is characterized by recurrent itchy hives, which can be associated with or without angioedema, or severe swelling under the skin.
What is Xolair?
Xolair, developed by Genentech and Novartis Pharmaceuticals, is approved in the U.S. and Europe for allergic asthma and CSU. But the therapy, a monoclonal antibody against immunoglobulin E (IgE), is also being investigated for angioedema. IgE is a protein involved in immune responses, including those at play in severe allergic reactions.
Data from previous Phase 3 trials in CSU patients, of whom up to 53% also had angioedema, showed that Xolair could also lessen angioedema symptoms.
However, evidence of its efficacy and safety in patients with idiopathic angioedema remains limited to a small number of case reports. Idiopathic angioedema is marked by chronic, recurrent swelling attacks that happen regularly without an identifiable cause.
Now, a team led by physicians at the University of Wisconsin, in Madison, together with Novartis conducted an exploratory Phase 4 trial (NCT02966314) to assess the safety and efficacy of Xolair for adults with idiopathic angioedema.
Participants had 12.9 angioedema episodes in the previous six months, on average.
In total, 10 patients — four women and six men — with a mean age of 53.4 years were randomly assigned to a placebo (five patients) or Xolair (five patients), given via monthly under-the-skin injections, for a total of 24 weeks (six months). Treatment was given as an add-on to standard therapy. After completing treatment, patients were followed for an additional 12 weeks.
this study provides preliminary prospective evidence that [Xolair] improves outcomes in patients with [idiopathic angioedema]
What did the study measure?
The study’s main goal was to assess the therapy’s effectiveness, as measured by changes in the mean Angioedema Activity Score for 7 days (AAS7), a measure of the frequency and intensity of angioedema episodes, since the study’s start (baseline). Higher scores indicate more active disease.
Secondary goals included evaluating changes in quality of life, assessed with the Angioedema Quality of Life questionnaire (AE-QoL), attack severity (assessed using a Visual Analogue Scale, VAS), and the number of angioedema episodes per month. In the AE-QoL, higher scores indicate worse quality of life, while higher scores in VAS indicate less severe symptoms.
Results showed that compared with placebo, Xolair lessened disease severity, and lowered the total number of angioedema episodes (56 vs. 7).
Compared with placebo, Xolair also improved quality of life and reduced the duration of angioedema episodes (5.48 vs. 1.23 hours).
Also, four of the five patients given Xolair seemed to achieve disease remission, as shown by a AAS7 score of zero and the maximum (100 score) in VAS.
Overall, “this study provides preliminary prospective evidence that omalizumab improves outcomes in patients with [idiopathic angioedema],” the researchers wrote.