The Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to ligelizumab (Novartis) for the treatment of chronic spontaneous urticaria in patients who have an inadequate response to H1-antihistamine treatment.

Ligelizumab is a high-affinity monoclonal anti-immunoglobulin E (IgE) antibody. The investigational drug is believed to work by inhibiting the IgE/FcεRI pathway, which plays a key role in the inflammatory process in chronic spontaneous urticaria (CSU). 

The designation is supported by data from a phase 2b dose-finding trial that compared the efficacy and safety of ligelizumab monthly subcutaneous injections to omalizumab (Xolair®; Genentech and Novartis) in CSU patients who have had an inadequate response to H1-antihistamines. Results showed that a greater proportion of patients treated with ligelizumab achieved complete resolution of wheals compared with those who received omalizumab. Moreover, ligelizumab was associated with rapid onset of action and sustained efficacy.

The Company is currently evaluating the efficacy and safety of ligelizumab in patients aged 12 years and older with CSU in the phase 3 PEARL 1 and PEARL 2 trials. Results from these trials are expected in the second half of 2021.

“Chronic spontaneous urticaria is a debilitating disease that may significantly impact a patient’s life,” said 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.”

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1.    Novartis ligelizumab (QGE031) receives FDA Breakthrough Therapy designation for patients with chronic spontaneous urticaria (CSU). [press release]. Basel, Switzerland: Novartis; January 14, 2021. 

2.    Novartis advances ligelizumab (QGE031) in urticaria to phase III on basis of strong phase II head-to-head data. [press release]. Basel, Switzerland: Novartis; December 4, 2018.