Ligelizumab from Novartis now has Breakthrough Therapy designation from FDA for the treatment of chronic spontaneous urticaria (CSU), also known as chronic idiopathic urticaria (CIU), in patients who have an inadequate response to H1-antihistamine treatment. 

Ligelizumab (QGE031) is currently being compared with omalizumab (Xolair® ) in an ongoing Phase 3 clinical trial programs (including PEARL 1 and PEARL 2) that have recruited more than 2,000 patients globally across 48 countries. Results are expected in the second half of 2021.

Ligelizumab is a next generation monoclonal anti-immunoglobulin E (IgE) antibody that is thought to work by blocking the IgE/FcεRI pathway, a key driver of the inflammatory process in CSU. In a Phase 2b dose-finding trial, more patients experienced complete resolution of wheals with ligelizumab, compared with omalizumab. No safety concerns were found with ligelizumab compared with omalizumab or placebo in a Phase 2b dose-finding trial in CSU patients with inadequate control on antihistamines. 

“Chronic spontaneous urticaria is a debilitating disease that may significantly impact a patient’s life. With so few treatment options available, patients are looking for more and better therapies to control their disease,” says Angelika Jahreis MD, PhD, Novartis Global Head Development Unit Immunology, Hepatology & Dermatology. “The FDA Breakthrough Therapy designation recognizes the need for a more effective treatment for this unpredictable, systemic and debilitating disease.” 

According to FDA guidelines, treatments that receive Breakthrough Therapy Designation must target a serious or life-threatening disease and demonstrate a potential substantial improvement over existing therapies on one or more significant clinical endpoints.